Historical Catalyst Calendar

Biotech Stock Historical Catalysts listed from 2009.

What's included?

2009 ~ Most small/mid capFDA Approval and Complete Response Letters.

January 2015 ~ Most key small/mid cap Phase 3 data releases.

August 2016 ~ Most key small/mid cap Phase 2 and Phase 3 data releases.

January 2016 ~ Most key large cap releases (data and regulatory).

View the BioPharmCatalyst FDA Calendar for a list of upcoming key catalysts.

Note: Most bankrupt or acquired companies are not included.

Key catalysts are generally inputted at the end of each trading session.

showing 2402 records
Ticker Drug Stage Catalyst
SPPI ZEVALIN
Non-Hodgkin’s lymphoma
 CRL
CRL received July 5, 2009.
ZGNX Sumavel
Migraine
 Approved
Approved July 16, 2009.
SPPI ZEVALIN
Non-Hodgkin’s lymphoma
 Approved
Approved September 4, 2009.
SPPI FUSILEV
Colorectal cancer
 CRL
CRL received October 9, 2009.
PRTK Intermezzo
Insomnia
 CRL
CRL issued November 2, 2009.
AIM Ampligen
CFS
 CRL
CRL issued December 1, 2009.
ACOR Ampyra
Improve walking in patients with multiple sclerosis
 Approved
Approved January 22, 2010.
ALKS BYDUREON
Type 2 diabetes
 Approved
Approved January 27, 2010.
ALKS BYDUREON
Type 2 diabetes
 CRL
CRL received March 15, 2010.
MNKD AFREZZA
Type 1/2 diabetes
 CRL
CRL issued March 15, 2010.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
 CRL
CRL issued March 19, 2010.
CTIC PIXUVRI - PIX306 Trial
B-cell non-Hodgkin lymphoma
 CRL
CRL received April 9, 2010.
JAZZ JZP-6
Fibromyalgia
 CRL
CRL received October 11, 2010.
VVUS Qnexa
Obesity
 CRL
October 28, 2010.
MNKD AFREZZA
Type 1/2 diabetes
 CRL
CRL received January 19, 2011.
ASRT GRALISE
Postherpetic neuralgia - shingles
 Approved
Approved January 28, 2011.
PLX Taliglucerase alfa
Gaucher disease
 CRL
CRL issued February 25, 2011.
SPPI FUSILEV
Colorectal cancer
 Approved
Approved April 29, 2011.
SGEN ADCETRIS
Hodgkin lymphoma and Anaplastic large cell lymphoma
 Approved
Approval announced August 19, 2011.
TAK ADCETRIS
Hodgkin lymphoma and Anaplastic large cell lymphoma
 Approved
Approval announced August 19, 2011.
PCRX EXPAREL
Postsurgical pain
 Approved
Approved October 31, 2011.
EYPT Iluvien
Diabetic macular edema
 CRL
CRL November 11, 2011.
ALIM Iluvien
Diabetic macular edema
 CRL
CRL November 2011.
INCY Jakafi
Myelofibrosis
 Approved
Approved November 16, 2011.
PRTK Intermezzo
Insomnia
 Approved
Approved November 23, 2011.
ATRS Anturol
Overactive bladder
 Approved
Approved December 8, 2011.
CRIS Vismodegib
Basal Cell Carcinoma
 Approved
Approved January 30, 2012.
CORT Corlux - Korlym
Cushing's Syndrome
 Approved
Approved February 17, 2012.
VVUS Avanafil
Erectile dysfunction
 Approved
Approved April 27, 2012.
PLX Taliglucerase alfa
Gaucher disease
 Approved
Approved May 1, 2012.
EBS BioThrax
Anthrax Vaccine
 Approved
Approved May 17, 2012.
RGEN RG1068
Detection of pancreatic duct abnormalities
 CRL
CRL received June 22, 2012.
ARNA Lorcaserin
Obesity
 Approved
Approved June 27, 2012.
VVUS Qnexa
Obesity
 Approved
Approved July 17, 2012.
AMRN Vascepa
High Triglycerides With Mixed Dyslipidemia
 Approved
Approved July 26, 2012.
HALO HYQVIA
Subcutaneous treatment for patients with primary immunodeficiency (PI)
 CRL
CRL issued August 1, 2012.
NAVB Lymphoseek
Lymphatic-tissue tracing agent
 CRL
CRL issued September 10, 2012.
SUPN Oxtellar
Epilepsy
 Approved
Approved October 22, 2012.
EXEL Cabozantinib
Medullary thyroid cancer
 Approved
Approved November 29, 2012.
TAK Cabozantinib
Medullary thyroid cancer
 Approved
Approved November 29, 2012.
AIM Ampligen
CFS
 CRL
CRL February 4, 2013.
IMGN Kadcyla
2nd-line HER2+ Metastatic Breast Cancer
 Approved
Approved February 22, 2013 - under priority review.
DVAX HEPLISAV-B
Hepatitis B
 CRL
CRL issued February 25, 2013.
NAVB Lymphoseek
Lymphatic-tissue tracing agent
 Approved
Approved March 13, 2013.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
 CRL
CRL March, 28 2013.
TTNP Probuphine
Opioid dependence
 CRL
CRL issued April 30, 2013.
ENDP AVEED (testosterone undecanoate) intramuscular injection
Men diagnosed with hypogonadism
 CRL
CRL issued September 5, 2013.
ASRT SEFELSA
Hot flashes
 CRL
CRL issued May 31, 2013.
AVDL Bloxiverz (neostigmine methylsulfate)
Reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery
 Approved
Approved June 3, 2013.
AVEO Tivozanib
Third line treatment of patients with renal cell cancer
 CRL
CRL issued June 10, 2013.
TBPH VIBATIV (telavancin hydrochloride) for injection
Hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP)
 Approved
Approved June 21, 2013.
SUPN Trokendi
Epilepsy
 Approved
Approved Aug 19, 2013.
ATRS OTREXUP
Rheumatoid arthritis (RA)
 Approved
Approved Oct 14, 2013.
ALIM Iluvien
Diabetic macular edema
 CRL
Second CRL October 17, 2013.
EYPT Iluvien
Diabetic macular edema
 CRL
Second CRL October 18, 2013.
ZGNX Zohydro ER
Moderate to severe pain
 Approved
Approved October 25, 2013.
ABBV Ibrutinib
Relapsed or refractory MCL mantle cell lymphoma
 Approved
Approved November 13, 2013.
AMAG Feraheme
Adults with iron deficiency anemia (IDA)
 CRL
CRL issued January 22, 2014.
VNDA HETLIOZ (tasimelteon)
Insomnia
 Approved
Approved January 31, 2014.
DRRX Posidur
Post-operative pain relief
CRL
CRL issued February 12, 2014.
ABBV Imbruvica
Cancer - Chronic Lymphocytic Leukemia Who Have Received at Least One Prior Therapy
 Approved
Approved February 12, 2014.
BMRN Vimizim (GALNS)
(MPS IVA) Morquio A Syndrome
 Approved
Approved February 14, 2014.
ENDP AVEED (testosterone undecanoate) intramuscular injection
Men diagnosed with hypogonadism
 Approved
Approved March 6, 2014.
NVS Cangrelor
Reduction of thrombotic cardiovascular events including stent thrombosis in patients with coronary artery disease undergoing percutaneous coronary intervention (PCI)
 CRL
CRL April 30 2014.
OMER Omidria
Cataract surgery
 Approved
Approved June 2, 2014.
BDSI BEMA Buprenorphine/Naloxone (BNX) - BUNAVAIL
Opioid dependence
 Approved
Approved June 6, 2014.
NAVB Lymphoseek
Head and Neck Cancer
 Approved
Approved June 13, 2014.
MNKD AFREZZA
Type 1/2 diabetes
 Approved
Approved June 27, 2014.
AVDL VAZCULEP (phenylephrine hydrochloride)
Clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia
 Approved
Approved June 30, 2014.
EGRX EP-3101 (bendamustine RTD)
Chronic lymphocytic leukemia; Indolent non-Hodgkin's lymphoma - cancer
 Approved
Tentative approval July 2 2014. Teva has also received orphan drug and related pediatric exclusivity expiring in September 2015 and May 2016 for the CLL and NHL indications, respectively.
SPPI Belinostat
Peripheral T-Cell Lymphoma cancer
 Approved
Approved July 7, 2014.
ABBV Imbruvica
Deletion 17p
 Approved
Approved July 19, 2014.
EGRX Ryanodex
Malignant hyperthermia - cancer
 Approved
Approved July 23, 2014 under priority review.
ACRX Zalviso
Post-operative pain following open abdominal surgery and hip or knee replacement surgery
 CRL
CRL issued July 25 2014.
NVS Oritavancin
ABSSSI
 Approved
Approved August 6, 2014 under priority review
AKBA Auryxia
Hyperphosphatemia
 Approved
Approved Sept 5, 2014.
HALO HYQVIA
Subcutaneous treatment for patients with primary immunodeficiency (PI)
 Approved
Approved September 12, 2014.
NKTR MOVANTIK (Naloxegol)
Opioid-induced constipation (OIC)
 Approved
Approved September 16, 2014.
EYPT Iluvien
Diabetic macular edema
 Approved
Approved Sept 26 2014.
ALIM Iluvien
Diabetic macular edema
 Approved
Approved September 26, 2014,
LNTH Subcutaneous RELISTOR
Chronic pain
 Approved
Approved September 29, 2014.
AEZS Macimorelin Acetate - Macrilen
Adult Growth Deficiency
 CRL
CRL issued November 6, 2014.
INCY Jakafi (ruxolitinib) (RESPONSE)
Polycythemia Vera
 Approved
Approved December 4, 2014.
ABBV VIEKIRA PAK
HCV - genotype 1
 Approved
Approved December 19, 2014.
ENTA VIEKIRA PAK
HCV - genotype 1
 Approved
Approved December 19, 2014.
BCRX RAPIVAB (peramivir)
INFLUENZA ACUTE
 Approved
Approved December 22, 2014.
ADMS Namzaric
Moderate to severe dementia of the Alzheimer's type.
 Approved
Approved December, 24 2014.
VRTX KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
 Approved
Approved December 29, 2014.
NBIX Elagolix
Endometriosis
 Phase 3
First Phase 3 trial met both co-primary endpoints - January 8, 2015.
ABBV Elagolix
Endometriosis
 Phase 3
First Phase 3 trial met both co-primary endpoints - January 8, 2015.
RMTI Soluble Ferric Pyrophosphate
Anemia
 Approved
Approved January 26, 2015.
ABBV IMBRUVICA
Waldenström’s Macroglobulinemia
 Approved
Approved January 29, 2015 - PCYC
ZGNX Abuse deterrent formulations of Zohydro ER
Moderate to severe pain
 Approved
Approved January 30, 2015.
PCRX EXPAREL
Post surgical pain
 CRL
CRL March 2, 2015.
VRTX KALYDECO (ivacaftor)
Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations
 Approved
Approved March 17, 2015.
NKTR Etirinotecan pegol NKTR-102 (BEACON)
Cancer - Metastatic Breast Cancer
 Phase 3
Phase 3 topline data mid March 17, 2015 did not reach primary endpoint.
ADMP Epinephrine Pre-filled Syringe (PFS)
Emergency treatment of anaphylaxis.
 CRL
CRL issued March 27 2015.
EBS Anthrax Immune Globulin Intravenous (Human) [AIGIV]
Anthrax
 PDUFA
Approved March 25, 2015.
AMGN Corlanor
Chronic Heart Failure
 Approved
Approved April 15, 2015.
JNJ Generic Copaxone
Multiple sclerosis
 Approved
Approved April 16 2015.
MNK RAPLIXA
Hemostasis
 Approved
Approved April 30, 2015.
TBPH BREO ELLIPTA
Asthma
 Approved
Approved April 30, 2015.
NVS IONSYS
Acute postoperative pain
 Approved
Approved April 30, 2015.
VRTX Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation
 Approved
Approved July 2, 2015.
NYMX NX-1207 Fexapotide
BPH
 Phase 3
Phase 3 endpoints met in extension trial July 2015.
SGEN ADCETRIS - AETHERA
Post-transplant Hodgkin lymphoma (HL) cancer
 Approved
Approved August 17, 2015 under priority review.
TAK ADCETRIS - AETHERA
Post-transplant Hodgkin lymphoma (HL) cancer
 Approved
Approved August 17, 2015 under priority review.
MACK ONIVYDE
Cancer - second line pancreatic
 Approved
Approved October 22, 2015.
IMGN Kadcyla GATSBY trial
Cancer - metastatic HER2+ gastric cancer
 Phase 3
Phase 3 endpoint not met - October 22, 2015.
SPPI EVOMELA (melphalan) for Injection
Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma
 CRL
CRL issued October 23, 2015.
BDSI BEMA Buprenorphine
Pain
 Approved
Approved October 26, 2015.
ENDP BEMA Buprenorphine
Management of pain severe enough to require daily, around-the-clock, long-term opioid treatment for which alternative treatment options are inadequate
 Approved
Approved October 26, 2015.
EXEL Vemurafenib
BRAF V600 Mutation-Positive Advanced Melanoma - Cancer
 Approved
Approved November 10, 2015.
NKTR ADYNOVATE
Hemophilia A
 Approved
Approved November 16, 2015.
EBS BioThrax
Anthrax Vaccine Adsorbed
 Approved
Approved November 24, 2015.
OTIC OTIPRIO (ciprofloxacin otic suspension)
Ear Tube Placement Surgery
 Approved
Approved December 11, 2015.
ADMS GOCOVRI (ADS-5102)
Levodopa-Induced Dyskinesia + Parkinson's disease patients receiving levodopa and experiencing OFF episodes
 Phase 3
Phase 3 data released December 23, 2015.
EGRX Docetaxel Injection
Breast, non-small cell lung, prostate gastric adenocarcinoma, and head and neck cancer
 Approved
Approved December 24, 2015.
CHRS CHS-0214
Rheumatoid arthritis
Phase 3
Phase 3 data released January 2016. Endpoints met. However, commercialization not possible in US before 2029 due to patent issues
BMRN Kyndrisa
Duchenne Muscular Dystrophy (DMD)
 CRL
CRL issued January 14 2016.
NEOS Adzenys XR-ODT
Attention deficit hyperactivity disorder (ADHD)
 Approved
Approved January 27, 2016.
VRTX KALYDECO (ivacaftor)
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
 CRL
CRL issued February 5, 2016.
NBIX Elagolix
Endometriosis
 Phase 3
First Phase 3 trial met both co-primary endpoints - Jan 2015. Data from second Phase 3 released February 2016 also met endpoints.
ABBV Elagolix
Endometriosis
 Phase 3
First Phase 3 trial met both co-primary endpoints - Jan 2015. Data from second Phase 3 released February 2016 also met endpoints.
RDHL Talicia RHB-105
H. pylori
 Phase 3
Phase 3 final data released March 8, 2016.
GWPH Epidiolex
Dravet Syndrome and Lennox-Gastaut syndrome
 Phase 3
Top line Phase 3 data released March 14, 2016 met primary endpoint.
SPPI EVOMELA (melphalan) for Injection
Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma
 Approved
Approved March 15 2016.
EGRX KANGIO (bivalirudin injection)
Percutaneous Coronary Intervention (PCI) and Percutaneous Transluminal Coronary Angioplasty (PTCA)
 CRL
CRL March 18 2016. FDA requested further characterization of bivalirudin-related substances in the drug product.
ALXN Bevyxxa (betrixaban)
Venous thromboembolism (VTE) Prevention
 Phase 3
Phase 3 data released March 24, 2016 did not meet primary endpoint.
OPK RAYALDEE
Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) and vitamin D insufficiency - Stage 3 and 4
 CRL
CRL issued March 30, 2016.
CHMA Mycapssa
Acromegaly
 CRL
CRL April 15, 2016.
EXEL CABOMETYX (cabozantinib)
Metastatic renal cell cancer (RCC)
 Approved
Approved April 25, 2016.
ACAD Pimavanserin
Parkinson’s disease psychosis (PDP)
 Approved
Approved April 29, 2016.
AVDL Akovaz
Hypotension
 Approved
Approved May 2, 2016.
TTNP Probuphine
Opioid dependence
 Approved
CRL issued April 30, 2013. Approved May 26, 2016.
VNDA Fanapt
Supplemental New Drug Application (sNDA) as a maintenance treatment of schizophrenia in adults.
 Approved
Approved May 26, 2016.
AZN Lokelma (ZS-9)
Hyperkalaemia
 CRL
CRL issued May 26, 2016.
ICPT Obeticholic acid (Ocaliva)
Primary biliary cirrhosis (PBC) - POISE
 Approved
Approved May 27 2016.
ADMP Epinephrine Pre-filled Syringe (PFS)
Emergency treatment of anaphylaxis.
 CRL
CRL issued March 27 2015. CRL issued again June 6, 2016.
OCUL DEXTENZA
Allergic conjunctivitis
 Phase 3
Phase 3 trial did not meet primary endpoint - June 6, 2016.
RVNC DAXI (RT002)
Lateral Canthal (Crow’s Feet) Lines
 Phase 3
Phase 3 endpoints not met June 13, 2016.
MRNS Ganaxolone
Focal onset seizures
 Phase 3
Phase 3 data did not meet endpoints - June 13, 2016
PRTK Omadacycline
Acute bacterial skin and skin structure infections (ABSSSI)
 Phase 3
Phase 3 top-line data met endpoints - June 16, 2016
OPK RAYALDEE
Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) and vitamin D insufficiency - Stage 3 and 4
 Approved
Announced FDA Approval June 21, 2016.
NVS Meropenem-vaborbactam
Complicated urinary tract infections (cUTI)
 Phase 3
Phase 3 data met endpoints June 27, 2016.
CLVS Rociletinib
Mutant EGFR T790M-positive lung cancer
 CRL
CRL June 28, 2016. Phase 3 TIGER-3 also terminated.
GSK Niraparib - NOVA (Niraparib Ovarian)
Cancer - ovarian
 Phase 3
Phase 3 data met endpoints - June 2016.
LPCN TLANDO - LPCN 1021
Men with low testosterone (Low T)
 CRL
CRL issued June 29, 2016.
AMPE Ampion
Osteoarthritis of the Knee
 Phase 3
STRIDE study mid April 2015 did not meet endpoint. Second Phase 3 trial also did not meet primary endpoint - June 2016
RARE MEPSEVII (vestronidase alfa)
Mucopolysaccharidosis 7 (MPS 7)
 Phase 3
Phase 3 data released July 2016. Primary endpoint hit. Secondary missed.
LNTH Oral RELISTOR
Chronic pain
 Approved
Approved July 19 2016.
OCUL DEXTENZA
Ocular inflammation and pain following cataract surgery
 CRL
CRL July 25 2016 - due to manufacturing deficiencies.
ADMA ASCENIV (RI-002)
Primary Immune Deficiency Diseases
 CRL
CRL issued July 29 2016. Third-party manufacturing issues cited.
MCRB SER-109 - ECOSPOR III
Recurrent C. Difficile infection
 Phase 2
Phase 2 interim data did not meet primary endpoint.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
 Phase 3
Phase 3 ENDEAR trial met primary endpoint August 1, 2016.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
 Approved
CRL Mar 28 2013. Approved August 10, 2016.
AUPH Voclosporin - AURORA
Lupus
 Phase 2b
Phase 2b data released August 2016. Primary endpoint met but with high number of deaths.
ACRX DSUVIA (ARX-04)
Moderate-to-severe acute pain following a surgical procedure
 Phase 3
Phase 3 data released August 15, 2016.
ALXN Andexxa
Factor Xa inhibitor reversal agent
 CRL
CRL August 17 2016.
ACRS A-101 THWART-1 and THWART-2
Common warts (verruca vulgaris)
 Phase 2
Phase 2 data released August 2016.
EARS AM-101 (Keyzilen) - TACTT2
Acute inner ear tinnitus
 Phase 3
Phase 3 trial did not meet primary endpoints.
KMDA Inhaled formulation of AAT
AATD - Alpha-1 Antitrypsin deficiency
 Phase 2
Top-line data released August 30, 2016. Primary endpoint met
RIGL Tavalissetm (Fostamatinib)
Immune Thrombocytopenic Purpura (ITP)
 Phase 3
Phase 3 data from the first trial released August 30 2016. Primary endpoint met.
GSK Rolapitant
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
 Approved
Approved September 2, 2015.
OPK Rolapitant
Prevention of chemotherapy induced nausea and vomiting, or CINV
 Approved
Approved September 2, 2015.
TNXP TNX-102 SL (Tonmya)
Fibromyalgia
 Phase 3
Phase 3 data released September 6, 2016. Primary endpoint not met. Program discontinued
KPTI Selinexor
Quadruple Refractory Multiple Myeloma
 Phase 2
Phase 2 positive top-line data released September 6, 2016
AERI Roclatan
Glaucoma
 Phase 3
Phase 3 Mercury 1 data released September 14, 2016. Primary endpoint met.
NVAX RSV vaccine - Resolve trial
Respiratory Syncytial Virus (RSV) in older adults
 Phase 3
Phase 3 released September 15, 2016 did not demonstrate efficacy.
SRPT Eteplirsen - Exon 51
Duchenne muscular dystrophy
 Approved
Approved September 19, 2016.
ITCI Lumateperone
Schizophrenia
 Phase 3
Phase 3 trial failed - September 28, 2016.
GNCA GEN-003
Genital herpes
 Phase 2b
Phase 2b virologic efficacy data released September 2016.
TENX Levosimendan
Septic shock
 Phase 2b
Phase 2b trial did not meet primary endpoint.
ALKS ALKS 5461
Major depressive disorder
 Phase 3
Phase 3 data from first two trials did not meet their endpoints. FORWARD-5 data, the third of three trials did meet endpoints - October 2016.
PLXP Locilex
Mild infections of diabetic foot ulcers
 Phase 3
Phase 3 top-line data did not meet endpoints - October 25, 2016 - TICKER CHANGED FROM DPRX FOLLOWING MERGER
RIGL Tavalissetm (Fostamatinib)
Immune Thrombocytopenic Purpura (ITP)
 Phase 3
Phase 3 data from second trial did not meet the primary endpoint.
AMAG Vyleesi (Bremelanotide)
Female sexual dysfunction (FSD)
 Phase 3
Phase 3 data released November 1, 2016.
PTN Vyleesi (Bremelanotide)
Female sexual dysfunction (FSD)
 Phase 3
Phase 3 data released November 1, 2016.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
 Phase 3
Phase 3 CHERISH data met primary endpoint - November 7, 2016.
DVAX HEPLISAV-B
Hepatitis B
 CRL
CRL November 14, 2016
ALNY Inclisiran
Hyperlipidemia
 Phase 2
Phase 2 Day 180 data presented at AHA Scientific Sessions on November 15, 2016.
NVS Inclisiran
Hyperlipidemia
 Phase 2
Phase 2 Day 180 data presented at AHA Scientific Sessions on November 15, 2016.
ACRS ESKATA (hydrogen peroxide)
Seborrheic keratosis (SK)
 Phase 3
Phase 3 trial met endpoints - November 15, 2016.
BSTC QWO
Edematous fibrosclerotic panniculopathy (“cellulite”)
 Phase 2b
Phase 2b data released November 17, 2016. Met endpoints
ENDP QWO
Edematous fibrosclerotic panniculopathy (“cellulite”)
 Phase 2b
Phase 2b data released November 17, 2016. Met endpoints
ACOR Dalfampridine
Post stroke deficits
 Phase 3
Phase 3 data released November 21, 2016 - failed to show efficacy, development to be discontinued.
BXRX Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery
 Phase 3
Phase 3 data from second trial released November 28, 2016. Primary endpoint met.
CERC CERC-301
Major depressive disorder (MDD)
 Phase 2
Phase 2 top-line data released November 29, 2016 failed to meet primary endpoint.
NOVN SB206
Human papillomavirus (HPV)
 Phase 2
Phase 2 data released November 29, 2016. Primary endpoint met.
HTBX HS-410
Bladder cancer
 Phase 2
Phase 2 trial did not meet primary endpoint - November 30, 2016.
HZNP ACTIMMUNE
Friedreich's Ataxia (FA)
 Phase 3
Phase 3 data released December 8, 2016 - primary endpoint not met
ISEE Fovista
Wet age-related macular degeneration (Wet-AMD)
 Phase 3
Phase 3 data released December 12, 2016 - primary endpoint not met.
VCEL MACI
Focal chondral cartilage defects in the knee.
 Approved
FDA Approval announced December 13, 2016 ahead of PDUFA date of January 3 2017.
BDSI Clonidine gel
Painful diabetic neuropathy (PDN)
 Phase 2b
Phase 2b data released December 13, 2016 - primary endpoints not met.
VTVT TTP273 - LOGRA
Type 2 Diabetes
 Phase 2
Phase 2 primary endpoint met - December 14, 2016.
CLVS Rucaparib ARIEL2
Cancer - ovarian second line
 Approved
Approved December 19, 2016. Original PDUFA date under priority review was February 23, 2017.
ACAD Pimavanserin - Harmony
Alzheimer’s disease psychosis
 Phase 2
Phase 2 top-line data released December 20, 2016. Primary endpoint met. Key secondary endpoint missed.
LXRX Sotagliflozin
Type 1 Diabetes
 Phase 3
Phase 3 data from second trial released December 21, 2016 - primary endpoint met.
MACK MM-302 HERMIONE
Cancer - HER2-positive locally advanced or metastatic breast cancer
 Phase 2
Phase 2 trial discontinued December 21, 2016 due to poor efficacy.
NVS Lutathera
Inoperable progressive midgut NETs
 CRL
PDUFA date under priority review was December 28, 2016. CRL issued December 21, 2016.
IRWD Linaclotide
Irritable Bowel Syndrome with Constipation (IBS-C)
 Phase 2b
Phase 2b data released December 22, 2016.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
 Approved
Approved December 23, 2016.
AMPH Primatene Mist (epinephrine inhalation aerosol)
Asthma
 CRL
CRL issued December 27, 2016.
OPK hGH-CTP
Adults - growth hormone deficiency
 Phase 3
Phase 3 data released December 30, 2016 - primary endpoint not met.
AGRX Twirla
Contraceptive patch
Phase 3
CRL issued 2013. Phase 3 top-line data released January 3, 2017 - poor data but intends to reflile NDA 1H 2017.
AEZS Macimorelin Acetate - Macrilen
Adult Growth Deficiency
 Phase 3
Phase 3 data released January 4, 2017 - trial failed.
HALO HALO-301
Pancreatic cancer
 Phase 2
Primary endpoint met.
GNCA GEN-003
Genital herpes
 Phase 2b
Phase 2b data released January 5, 2017.
OTIC OTIPRIO
Acute otitis externa
 Phase 3
Phase 3 trial met primary endpoint - January 5, 2017.
CHRS CHS-1420
Psoriasis
 Phase 3
Phase 3 top-line data met endpoints August 2016. Full data due released January 10, 2016.
GSK Rolapitant - intravenous (IV)
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
 CRL
CRL issued January 11 2017.
FGEN Pamrevlumab (LAPIS)
Pancreatic cancer
 Phase 2
Phase 2 abstract released January 17, 2017.
NBIX INGREZZA
Tourette syndrome - adults
 Phase 2
Phase 2 data released January 18, 2017 did not meet primary endpoint.
ABBV Oxymetazoline HCl cream 1.0%
Facial Erythema (Redness) Associated with Rosacea
 Approved
Approved January 19, 2017.
ABBV Imbruvica
Marginal zone lymphoma
 Approved
sNDA filing under priority review approved January 19, 2017.
MRNS Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)
 Phase 2
Phase 2 preliminary CDKL5 cohort data released January 23, 2017.
LGND Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)
 Phase 2
Phase 2 preliminary CDKL5 cohort data released January 23, 2017.
IRWD Linaclotide 72 mcg dose for use in the treatment of adults
Chronic idiopathic constipation (CIC)
 Approved
sNDA approval announced January 26, 2017.
NOVN SB204
Acne vulgaris
 Phase 3
Phase 3 top-line data released January 27, 2017. Met one of three co-primary endpoints in NI-AC301 trial. Met all endpoints in NI-AC302 trial.
NRBO Gemcabene - COBALT-1
Homozygous Familial Hypercholesterolemia (HoFH)
 Phase 2b
Phase 2b interim data released January 30, 2017.
TENX Levosimendan
Reduce the incidence of low cardiac output syndrome during cardiac surgery
 Phase 3
Phase 3 top line data released January 31, 2017 - primary endpoints not met.
CATB Edasalonexent (CAT-1004)
Duchenne muscular dystrophy (DMD)
 Phase 1/2
Phase 1/2 top-line data released January 31 - primary endpoint not met.
GWPH Sativex (Nabiximols)
Spasticity due multiple sclerosis
 Phase 2
Phase 2 trial did not meet primary endpoint - noted February 7, 2017.
AMGN Parsabiv
Secondary hyperparathyroidism (SHPT)
 Approved
Approved February 7, 2017.
JNJ XARELTO (rivaroxaban)
Coronary Artery Disease or Peripheral Artery Disease
 Phase 3
Phase 3 trial stopped early due to positive efficacy - February 8, 2017.
ACOR INBRIJA (CVT-301)
OFF episodes of Parkinson’s disease (PD)
 Phase 3
Phase 3 data released February 9, 2017 - primary endpoint met.
TCON TRC105 and Avastin
Glioblastoma - cancer
 Phase 2
Phase 2 data released February 9, 2017 - primary endpoint not met.
ZSAN M207
Migraine
 Phase 3
Phase 3 trial data due released February 13, 2017 - primary endpoints met.
SAGE SAGE-217 - MOUNTAIN
Major Depressive Disorder (MDD)
 Phase 2
Phase 2 top-line data from open-label trial released February 13, 2017
MRK MK-8931 (017) - Verubecestat
Mild-to-moderate Alzheimer's
 Phase 3
Phase 3 trial stopped due to lack of efficacy - February 14, 2017.
BHC Brodalumab
Psoriasis
 Approved
Approved February 15, 2017.
ARDX Tenapanor
Serum phosphorus - chronic kidney disease (CKD) on dialysis
 Phase 3
Phase 3 released February 15, 2017 - primary endpoint met.
AZN Lynparza
Breast cancer
 Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met.
MRK Lynparza
Breast cancer
 Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met.
TRVN Oliceridine (TRV130)
Moderate to severe acute pain
 Phase 3
Phase 3 APOLLO-1 and APOLLO-2 data released February 21, 2017 - primary endpoints met but commercial doubts raised.
CDTX CD101 Topical - RADIANT
Vulvovaginal candidiasis (VVC)
 Phase 2
Phase 2 data released February 21, 2017 - efficacy not evident, Development to be discontinued.
AMPH Naloxone Intranasal
Opioid overdose
CRL
CRL announced February 21, 2017.
BMY REVLIMID
Multiple myeloma (MM) - autologous stem cell transplant (ASCT)
 Approved
Approved February 22, 2017.
CMRX Brincidofovir - Advise trial
Adenovirus Infection
 Phase 3
Phase 3 interim analysis released May 2016. Final data released February 22, 2017.
BCRX Berotralstat BCX7353
Reduce or eliminate attacks in HAE patients
Phase 2
Phase 2 interim data released February 27, 2017.
LJPC GiaprezaTM (angiotensin II)
Increase blood pressure in adults with septic or other distributive shock
 Phase 3
Phase 3 data released February 27, 2016 - primary endpoint hit. Secondary endpoints not mentioned. NDA filing due 2H 2017.
AZN Saxagliptin and dapagliflozin
Type-2 diabetes
 Approved
Approval announced February 28, 2017.
AUPH Voclosporin - AURORA
Lupus
 Phase 2b
Phase 2b data released August 2016. Primary endpoint met but with high number of deaths. Further data released March 1, 2017 - no additional deaths and endpoints met.
MRK Odactra (MK-8237)
House dust mite allergies
 Approved
Approved March 1, 2017.
PTCT Translarna
Nonsense mutation cystic fibrosis
 Phase 3
Phase 3 data released March 2, 2017 - endpoints not met. Development to be discontinued.
KPTI Selinexor SOPRA
Cancer - Relapsed/Refractory Acute Myeloid Leukemia (AML)
 Phase 2
Data Safety Monitoring Board (DSMB) noted March 2, 2017 that trial will not meet primary endpoint.
IONS Volanesorsen - APPROACH
Familial chylomicronemia syndrome (FCS)
 Phase 3
Phase 3 data released March 6, 2017 - primary endpoint met. Relative safety concerns noted.
KURA Tipifarnib - (AIM-HN/SEQ-HN)
Head and neck squamous cell carcinomas (HNSCC)
 Phase 2
Phase 2 ongoing data released March 6, 2017 - further data due 2H 2017.
TGTX TG-1101 and IMBRUVICA (GENUINE trial)
Chronic Lymphocytic Leukemia (CLL) cancer
 Phase 3
Phase 3 trial met primary endpoint - March 6, 2017, meeting with FDA in 2H 2017.
ACOR Ampyra
Improve walking in patients with multiple sclerosis
 -
Decision on Inter Partes Review (IPR) released March 10, 2017 - patents upheld.
NVS LEE011
HR+/HER2- advanced breast cancer
 Approved
Priority review granted November 1, 2016. Approval announced March 13, 2017.
MRK KEYTRUDA
Relapsed or Refractory Classical Hodgkin Lymphoma
 Approved
Approval announced March 14, 2017.
AZN Lynparza - SOLO 2
Second-line ovarian cancer
 Phase 3
Data presented March 14, 2017 showed significant PFS benefit.
ENDP OPANA ER
Management of pain
 -
Advisory Committee Meeting March 14, 2017 voted 18 to eight that the benefits of reformulated OPANA ER no longer outweigh its risks.
CPRX Firdapse
MuSK-antibody positive myasthenia gravis (MuSK-MG)
 Phase 2
Phase 2 top-line data met endpoints - March 15, 2017. Pivotal trial planned.
AZN Lokelma (ZS-9)
Hyperkalaemia
 CRL
Second CRL issued March 17, 2017.
AMGN Repatha
Cardiovascular disease
 Phase 3
Phase 3 cardiovascular outcomes study data released March 17, 2017 - endpoints met but down on expectations.
LLY Abemaciclib - MONARCH 1 and 2
Second-line hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer and HR+, HER2- breast cancer
 Phase 3
Phase 3 data released March 20, 2017 - PFS primary endpoint met.
BLRX BL-8040 GENESIS
Stem-cell mobilization for autologous transplantation
 Phase 2
Phase 2 trial initiated March 2016. Partial data released March 20, 2017.
JAZZ JZP-110
Excessive sleepiness associated with obstructive sleep apnea
 Phase 3
Phase 3 data released March 20, 2017 - primary endpoints met.
NVS RELAX-AHF-2 (RLX030)
Acute heart failure
 Phase 3
Phase 3 data released March 22, 2017 - primary endpoints not met.
PFE Avelumab
Metastatic Merkel cell carcinoma
 Approved
Priority review announced November 29, 2016. Approval announced March 23, 2017.
XENE XEN801
Acne
 Phase 2
Phase 2 data released March 24 2017 - endpoints not met.
GSK Niraparib - NOVA (Niraparib Ovarian)
Cancer - ovarian
 Approved
PDUFA date under priority review was June 30, 2017. Approved March 27, 2017.
MNLO FMX101
Acne
 Phase 3
Phase 3 data released March 27, 2017 - three of four primary endpoints met across two trials.
PRTK Sarecycline
Severe acne vulgaris
 Phase 3
Phase 3 data released March 27 2017 - primary endpoints met. NDA filing due 2H 2017.
VRTX Tezacaftor / ivacaftor
Cystic fibrosis - Two Copies of the F508del Mutation
 Phase 3
Phase 3 data released March 28, 2017 - primary endpoint met. NDA filing due 3Q 2017.
QLGN RP-G28
Lactose intolerance
 Phase 2/3
Phase 2/3 top-line data released March 28, 2017. Primary endpoint met. End of Phase 2 meeting with FDA planned.
REGN Dupixent (dupilumab)
Atopic dermatitis
 Approved
Approved under priority review - March 28, 2017.
SNY Dupixent (dupilumab)
Atopic dermatitis
 Approved
Approved under priority review - March 28, 2017.
LNTH Azedra
Pheochromocytoma
 Phase 2b
Pivotal Phase 2 trial resumed January 2015 following suspension of trial in 2010 due to lack of funding. Primary endpoint met - March 30, 2017. NDA filing due mid-2017.
CRBP Lenabasum
Cystic Fibrosis
 Phase 2
Phase 2 data released March 30, 2017 - primary endpoint met but some concerns regarding efficacy.
AZN Tagrisso
Epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC)
 Approved
Approval announced March 31, 2017.
NVCR Tumor Treating Fields (TTFields) - INNOVATE-3
Ovarian cancer
 Phase 2
Phase 2 data presented December 12, 2016. Further promising PFS data released March 31, 2017.
PFE IBRANCE (palbociclib)
HR+, HER2- Metastatic Breast Cancer
 Approved
sNDA acceptance announced December 21, 2016. Approval announced March 31, 2017.
PRTK Omadacycline
Community-acquired bacterial pneumonia (CABP)
 Phase 3
Phase 3 data released April 3, 2017 - all endpoints met.
KPTI Selinexor
Diffuse Large B-Cell Lymphoma (DLBCL)
 Phase 2b
Phase 2b additional interim data released April 4, 2017 at AACR. Final top-line data due mid-2018.
NKTR Cipro DPI
Bronchiectasis
 Phase 3
Phase 3 second trial did not meet endpoint - April 5, 2017.
SUPN Trokendi
Migraine
 Approved
Tentative approval August 19 2016, subject to the pediatric exclusivity which expires March 28, 2017. Final approval announced April 6, 2017.
MRK Januvia (Sitagliptin)
Type 2 Diabetes
 CRL
CRL issued April 7 2017. sNDA for approved drug requested to include data on cardiovascular effects
GERN Imetelstat
Myelofibrosis
 Phase 2/3
Phase 2 clinical hold lifted Nov 2014. Phase 2 initiated September 2015. Internal review conducted September 2016. Low dose discontinued, high dose enrollment suspended. Further review conducted April 2017 - further development pending FDA feedback and longer term data.
MESO Rexlemestrocel
Heart failure
 Phase 3
Noted April 10, 2017 that interim analysis has been completed - trial to continue as planned.
NBIX INGREZZA
Tardive dyskinesia
 Approved
Approved April 11, 2017.
NOVN SB208
Fungal infections
 Phase 2
Phase 2 data released April 12, 2017. Primary endpoint met.
INCY Baricitinib
Rheumatoid arthritis
 CRL
Announced January 19 2016 that NDA Filing had been submitted. Three month delay announced January 13, 2017. CRL received April 14, 2017.
LLY Baricitinib
Rheumatoid arthritis
 CRL
Announced January 19 2016 that NDA Filing had been submitted. Three month delay announced January 13, 2017. CRL received April 14, 2017.
RARE Crysvita (Burosumab)
X-Linked Hypophosphatemia (XLH)
Phase 3
Phase 3 data released April 18, 2017 - primary endpoint met, two of three secondary endpoints met (missed on pain).
ABBV Veliparib
Squamous non-small cell lung cancer (NSCLC) and triple negative breast cancer (TNBC)
 Phase 3
Phase 3 trial did not meet primary endpoints - April 19, 2017.
MRK RENFLEXIS - SB2 (infliximab biosimilar)
Biosimilar candidate of Remicade
 Approved
BLA acceptance announced May 23, 2016 by partner Samsung Bioepis. Approval annuonced April 21, 2017.
EIGR Lonafarnib - D-LIVR
Chronic hepatitis delta virus (HDV)
 Phase 2
Phase 2 end of trial data released April 21, 2017. Meeting with FDA scheduled for 4Q 2017.
LLY Abemaciclib - MONARCH 3
HR+, HER2-locoregionally recurrent or metastatic breast cancer (MBC)
 Phase 3
Phase 3 interim analysis April 24, 2017 met primary endpoint. Detailed data due 2H 2017 with regulatory filings due 3Q 2017.
JAZZ Xyrem
Pediatric narcolepsy patients with cataplexy
 Phase 2/3
Phase 2/3 positive data released April 24, 2017. sNDA filing due 4Q 2017.
XOMA XOMA 213
Hyperprolactinemia
 Phase 2
Phase 2 proof-of-concept results released April 24, 2017.
AKTX Nomacopan
Paroxysmal nocturnal hemoglobinuria (PNH)
 Phase 2
Phase 2 interim data released April 24, 2017. Phase 3 program to commence 4Q 2017 with initial data due 1Q 2019.
REGN Praluent (alirocumab)
High low-density lipoprotein (LDL) cholesterol
 Approved
sBLA Approval announced April 25, 2017.
SNY Praluent (alirocumab)
High low-density lipoprotein (LDL) cholesterol
 Approved
sBLA Approval announced April 25, 2017.
JAZZ Solriamfetol (JZP-110)
Excessive sleepiness associated with narcolepsy
 Phase 3
Phase 3 data released April 26, 2017. NDA to be filed late 2017.
BMRN Cerliponase alfa
Batten Disease
 Approved
PDUFA date extended by three months to April 27 2017. Approval announced April 27, 2017.
NVS Rydapt (Midostaurin - PKC412)
Acute myeloid leukemia (AML)
 Approved
Priority review granted November 14, 2016. Approval announced April 28, 2017.
TGTX TG-1101 - ULTIMATE I
Multiple Sclerosis
 Phase 2
Phase 2 preliminary results presented at American Academy of Neurology (AAN) annual meeting April 28, 2017.
RDUS TYMLOS (Abaloparatide)
Osteoporosis
 Approved
PDUFA date originally scheduled for March 30 2017 delayed by three months to June 30, 2017. Approval announced April 28, 2017.
AEZS Zoptrex
Endometrial cancer
 Phase 3
Phase 3 trial did not meet primary endpoint - May 1, 2017.
AZN Urothelial carcinoma - Bladder cancer
Bladder cancer
Approved
BLA acceptance announced December 9, 2017. PDUFA under priority review. Approval announced May 1, 2017.
NTRP Bryostatin
Alzheimer's disease
 Phase 3
Phase 2 data released May 1, 2017 - primary endpoint not met.
AMAG Feraheme
Adults with iron deficiency anemia (IDA)
 Phase 3
Phase 3 trial completion announced May 2, 2017 - endpoints met. sNDA due mid-2017.
REGN Dupixent (dupilumab)
Eosinophilic esophagitis
 Phase 2
Phase 2 data released May 4, 2017.
SNY Dupixent (dupilumab)
Eosinophilic esophagitis
 Phase 2
Phase 2 data released May 4, 2017.
TXMD Yuvvexy - TX-004HR
Moderate-to-severe vaginal pain
 CRL
CRL announced May 8, 2017. Company noted that FDA cited a lack of long-term endometrial safety data.
PFE Avelumab
Urothelial Carcinoma
 Approved
Approval announced May 9, 2017.
LCTX OpRegen
Dry age-related macular degeneration (AMD)
 Phase 2
Phase 2 second cohort underway. Interim data presented at May 8, 2017 at ARVO conference.
MRK Keytruda
First-Line Treatment of Metastatic Non-Squamous Non-Small Cell Lung Cancer
Approved
Approved May 10, 2017.
GSK Mepolizumab
Chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype
 Phase 3
Phase 3 METREX trial met primary endpoint. Phase 3 METREO failed to meet endpoint.
AZN Tralokinumab (STRATOS1)
Severe, uncontrolled asthma
 Phase 3
Phase 3 trial did not meet primary endpoint - May 10, 2017.
LLY Galcanezumab - EVOLVE-1, EVOLVE-2 and REGAIN
Migraine prevention
 Phase 3
Phase 3 data released May 12, 2017 - primary endpoints hit in all trials. BLA filing due 2H 2017.
CAPR CAP-1002 ALLSTAR
Myocardial infarction (heart attack)
 Phase 2
Phase 2 data released May 12, 2017 - primary endpoint unlikely to be met.
AZN Durvalumab (PACIFIC)
Lung cancer
Phase 3
Phase 3 interim analysis released May 12, 2017 - PFS primary endpoint already met. OS endpoint to be assessed.
ARDX Tenapanor (T3MPO-1)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data released May 12, 2017. Primary endpoint met - however, competitive concerns raised.
IONS Inotersen (IONIS-TTRRx)
NEURO-TTR - familial amyloid polyneuropathy (FAP).
 Phase 3
Phase 3 data released May 15, 2017 - primary endpoint met but with safety concerns.
RVNC DAXI (RT002) - ASPEN
Cervical dystonia
 Phase 2
Phase 2 additional data released May 18, 2017 with oral presentation June 7, 2017 at International Congress of Parkinson's Disease and Movement Disorders.
MRK KEYTRUDA
First and Second line locally advanced or metastatic urothelial cancer - bladder cancer
 Approved
Approved May 18, 2017.
BMY ZEPOSIA (Ozanimod)
Relapsing Multiple Sclerosis
 Phase 3
Phase 3 data due released May 22, 2017 - primary endpoint met, disability endpoint not met.
AMGN EVENITY (Romosozumab)
Osteoporosis
 Phase 3
PDUFA date July 19, 2017. However, likely to be delayed due to safety data from ARCH trial released May 22, 2017.
SNY Sarilumab
Rheumatoid arthritis
 Approved
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
REGN Sarilumab
Rheumatoid arthritis
 Approved
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
LJPC GiaprezaTM (angiotensin II)
Increase blood pressure in adults with septic or other distributive shock
 Phase 3
Phase 3 data released February 27, 2016 - primary endpoint hit. Secondary endpoints not mentioned. Data released May 22, 2017 confirmed primary endpoint was met but with lack of mortality improvement.
MRK KEYTRUDA
Microsatellite Instability-High Cancer
 Approved
PDUFA date March 8, 2017 extended to June 9, 2017 due to submission of extra data. Approved May 23, 2017.
NBIX INGREZZA - T-Force GOLD
Tourette syndrome - juvenile
 Phase 2
Phase 2 data released May 23 - primary endpoint not met.
AERI Roclatan
Glaucoma
 Phase 3
Phase 3 Mercury 1 data released September 14, 2016. Primary endpoint met. Mercury 2 trial data released may 24, 2017 - primary endpoint also hit with NDA filing due 1H 2018.
PBYI Neratinib (NERLYNX)
Extended adjuvant HER2-positive early stage breast cancer
 -
Announced acceptance of NDA Filing September 20, 2016. PDUFA estimate July 20, 2017. Advisory Committee Meeting May 24, 2017 voted 12-4 in favor of recommending approval.
BCRX Berotralstat BCX7353
Reduce or eliminate attacks in HAE patients
Phase 2
Phase 2 interim data released February 27, 2017. Top-line data released May 25, 2017.
NVS Zykadia
First-line use in patients with ALK+ metastatic NSCLC
 Approved
Priority review granted February 22, 2017. Approval announced May 26, 2017.
MRK ISENTRESS
HIV-1
 Approved
Approval announced May 30, 2017.
GILD Bictegravir/F/TAF
HIV
 Phase 3
Phase 3 data released May 30 - primary endpoint met. NDA filing due 2Q 2017.
NVO Nonacog beta pegol
haemophilia B
 Approved
Approval announced May 31, 2017.
RETA Omaveloxolone
Friedreich’s ataxia (FA)
 Phase 2
Phase 2 initial data released June 1, 2017. Primary endpoint not met.
LLY Larotrectinib (LOXO-101)
Solid tumors that harbor a TRK fusion.
 Phase 2
Announced 76% ORR at ASCO - June 3, 2017. Primary analysis by the end of 2017 with NDA filing late 2017 or early 2018.
JNJ ZYTIGA (abiraterone acetate) - LATITUDE
metastatic hormone-naïve prostate cancer (mHNPC)
 Phase 3
Phase 3 data presented at ASCO June 3, 2017 - PFS 33 months.
TGTX TG-1101 and IMBRUVICA (GENUINE trial)
Chronic Lymphocytic Leukemia (CLL) cancer
 Phase 3
Phase 3 trial met primary endpoint - March 6, 2017. Data presented at ASCO 2017. Meeting with FDA in 2H 2017.
HALO HALO-301
Pancreatic cancer
 Phase 2
Phase 2 data released January 5, 2017. Oral presentation of Phase 2 data at ASCO June 4, 2017 showed ORR of 45%.
AZN Lynparza
Breast cancer
 Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met. Late breaker at ASCO June 4, 2017 showed HR of 0.58 (42% reduction of risk of disease progression or death).
MRK Lynparza
Breast cancer
 Phase 3
Phase 3 data released February 17, 2016 - primary endpoint met. Late breaker at ASCO June 4, 2017 showed HR of 0.58 (42% reduction of risk of disease progression or death).
FPRX Cabiralizumab (FPA008)
Pigmented Villonodular Synovitis (PVNS)
 Phase 1/2
Phase 1/2 initial data released at ASCO June 4, 2017.
PIRS PRS-080
Anemia of chronic disease
 Phase 1b
Phase 1b data released at ERA-EDTA June 4, 2017.
BLUE ide-cel bb2121 - KarMMa
Relapsed/refractory multiple myeloma
 Phase 1
Phase 1 interim data released November 30, 2016 - 78% ORR. Pivotal trial to be initiated 2017. Updated data at ASCO June 5, 2017 - ORR 89%.
BMY ide-cel bb2121 - KarMMa
Relapsed/refractory multiple myeloma
 Phase 1
Phase 1 interim data released November 30, 2016 - 78% ORR. Pivotal trial to be initiated 2017. Updated data at ASCO June 5, 2017 - ORR 89%.
GILD Axicabtagene ciloleucel - (ZUMA-3)
Adult patients with r/r ALL - cancer
 Phase 1
Phase 2 data due 2018. Phase 1 data released at ASCO June 5, 2017. Complete remission 73%.
IOVA LN-144 - lifileucel
Refractory metastatic melanoma
 Phase 2
Phase 2 second cohort commenced enrollment - May 19, 2017. Initial data presented at ASCO June 5, 2017.
CLSN GEN-1 OVATION 2
Ovarian cancer
 Phase 1b
Phase 1b interim data released at ASCO 2017 - ORR 86%.
BMY liso-cel (JCAR017)
Relapsed or refractory (R/R) large B-cell lymphoma
 Phase 1/2
Pivotal trial to be initiated in 2017. ASCO Poster Session June 5, 2017 - ORR 86%.
CRVS CPI-444
Solid tumors - cancer
 Phase 1b
Phase 1/1b preliminary data released November 2016. Further update at ASCO June 5, 2017.
GLYC Uproleselan (GMI-1271)
Acute myeloid leukemia (AML) newly diagnosed and refractory - cancer
 Phase 2
Phase 2 enrollment completed - announced May 31, 2017. Data updates at ASCO June 5, 2017.
ALKS Aristada
Schizophrenia
 Approved
Approved October 5 2015. sNDA filed for 2-month option with approval announced June 6, 2017.
NVS Kymriah (CTL019 )- JULIET
Cancer - Diffuse Large B-Cell Lymphoma (DLBCL)
 Phase 2
Phase 2 pivotal primary analysis due International Conference on Malignant Lymphoma (ICML) on June 14, 2017. Abstract released June 7, 2017. ORR 45%.
LXRX Sotagliflozin
Type 1 Diabetes
 Phase 3
Phase 3 data released September 9, 2016 met primary endpoint. Secondary endpoint data due May 2017. Data from second trial released December 21, 2016 - primary endpoint met. Data from third trial also met primary endpoint - June 9, 2017.
JNJ INVOKANA CANVAS
Type 2 diabetes
 Phase 3
Cardiovascular Outcomes trial data released June 12, 2017 - 14% reduction in the risk of the composite primary endpoint of CV mortality.
XLRN Dalantercept - DART Study
Renal Cell Carcinoma (RCC) - cancer
 Phase 2
Phase 2 data released June 12, 2017 - primary endpoint not met.
CHRS CHS-1701
Pegfilgrastim biosimilar
 CRL
CRL announced June 12, 2017.
EPZM Tazemetostat
Follicular lymphoma
 Phase 2
Phase 2 data released June 14, 2017 - DLBCL ORR 29%.
ALDX Reproxalap (ADX-102)
Allergic conjunctivitis
 Phase 2b
Phase 2b data released June 14 2017 - primary endpoint not met. However, Phase 3 trial is planned.
RDHL BEKINDA
Gastroenteritis
 Phase 3
Phase 3 top-line data released June 14, 2017 - primary endpoint met.
KURA Tipifarnib
Relapsed or refractory peripheral T-cell lymphoma
 Phase 2
Phase 2 data presented at ICML conference on June 14, 2017 - 3/18 partial responses.
ADMP Epinephrine Pre-filled Syringe (PFS)
Emergency treatment of anaphylaxis.
 Approved
CRL issued March 27 2015. CRL issued again June 6, 2016. Approval announced June 15, 2017 following third submission.
SELB SEL-212 (COMPARE) - Head to head trial vs Krystexxa
Tophaceous gout
 Phase 2
Phase 2 dosing commenced late October 2016. Initial data released at EULAR June 15, 2017.
ALXN Cerdulatinib (PRT2070)
Refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia - cancer
 Phase 2
Phase 2 enrollment commenced May 2016. Interim data released June 15, 2017 - noted CLL/SLL 67% partial responese rate + two patient deaths.
JNJ DARZALEX (Daratumumab) + pomalidomide and dexamethasone
Relapsed or refractory multiple myeloma
 Approved
Approval announced June 16, 2017 - note: submitted by Janssen Biotech, Inc.
GMAB DARZALEX (Daratumumab) + pomalidomide and dexamethasone
Relapsed or refractory multiple myeloma
 Approved
Approval announced June 16, 2017 - note: submitted by Janssen Biotech, Inc.
CLVS Rucaparib ARIEL3
Ovarian cancer patients with a BRCA-like mutation
 Phase 3
Phase 3 data released June 20 2017 - primary endpoint met. sNDA filing due 3Q or early 4Q 2017.
SGEN Vadastuximab Talirine (CASCADE)
Acute myeloid leukemia (AML) - cancer
 Phase 3
Phase 3 trial discontinued due to higher rate of deaths - June 19, 2017.
NEOS Cotempla XR-ODT
Attention deficit hyperactivity disorder (ADHD)
 Approved
Approval announced June 19, 2017.
LPCN TLANDO - LPCN 1021
Men with low testosterone (Low T)
 -
CRL issued June 2016. Flexibility trial data announced June 19, 2017 - primary endpoint met with NDA fiing due 3Q 2017.
NVS RTH258 (brolucizumab)
Wet age-related macular degeneration (AMD)
Phase 3
Phase 3 data released June 20, 2017 - primary and key secondary endpoints met.
CARA IV CR845
Acute pain
 Phase 3
Interim conditional power analysis conducted June 21, 2017 - trial completion due 4Q 2017.
PFE Epoetin alfa biosimilar
CRL
CRL issued June 22, 2017.
NVS Tafinlar (dabrafenib) and Mekinist (trametinib)
Non-small cell lung cancer (NSCLC) with BRAF V600E mutation
 Approved
FDA Approval announced June 22, 2017.
HALO Subcutaneous rituximab
Various blood cancers
 Approved
Approval announced June 22, 2017.
XOMA ACZ885 (canakinumab)
CV risk reduction
 Phase 3
Phase 3 trial met primary endpoint - data to be presented later in 2017 at a medical congress.
NVS ACZ885 (canakinumab)
CV risk reduction
 Phase 3
Phase 3 trial met primary endpoint - data to be presented later in 2017 at a medical congress.
GBT GBT440 HOPE-KIDS 1
Sickle cell disease - children age 6-17.
 Phase 1/2
Phase 2a initiated June 2016. Preliminary data released at European Hematology Association (EHA) - June 23, 2017.
BLCM BPX-501 Rivo-cel
Adjunct T-cell therapy administered after allogeneic HSCT - (hematopoietic stem cell transplantation) - cancer
 Phase 1/2
Phase 1/2 update June 23, 2017.
BLUE LentiGlobin - HGB-207 Northstar-2
non-β0/β0 transfusion-dependent thalassemia (TDT)
 Phase 3
Phase 3 early data released at EHA June 23, 2017.
ALXN Bevyxxa (betrixaban)
Venous thromboembolism (VTE) Prevention
 Approved
FDA Approval noted June 23, 2017.
AGIO AG-348 (ACTIVATE-T)
Pyruvate kinase deficiency
 Phase 1/2
Phase 2 updated data released at EHA June 24, 2017.
AMAG Makena - auto injector
Reduce the risk of preterm birth in women with a singleton pregnancy
 Phase 2
sNDA filing announced April 17, 2017. PDUFA date February 14, 2018. 10-month review despite initial expectations of a 6-month review.
ATRS Makena - auto injector
Reduce the risk of preterm birth in women with a singleton pregnancy
 Phase 2
sNDA filing announced April 17, 2017. PDUFA date February 14, 2018. 10-month review despite initial expectations of a 6-month review.
SGEN ADCETRIS in combination with chemotherapy ECHELON-1
Frontline Hodgkin lymphoma
 Phase 3
Phase 3 data released June 26, 2017 - primary endpoint met.
TAK ADCETRIS in combination with chemotherapy ECHELON-1
Frontline Hodgkin lymphoma
 Phase 3
Phase 3 data released June 26, 2017 - primary endpoint met.
XENE TV-45070
Postherpetic neuralgia
 Phase 2b
Phase 2b data released June 27, 2017. Endpoints not met.
NRBO Gemcabene - COBALT-1
Homozygous Familial Hypercholesterolemia (HoFH)
 Phase 2b
Phase 2b data released June 28, 2017 - primary endpoint met.
AMGN Vectibix (Panitumumab)
Wild-Type RAS Metastatic Colorectal Cancer
 Approved
sBLA approval announced June 29, 2017.
ALKS ALKS 3831 - ENLIGHTEN-1
Schizophrenia
 Phase 3
Phase 3 preliminary data released June 30, 2017. Primary endpoint met - adverse events included weight gain.
CARA CR845
Osteoarthritis (OA)
Phase 2b
Phase 2b data released June 29, 2017 - trial failed.
BMY ORENCIA
Psoriatic Arthritis (PsA)
 Approved
Approval announced July 6, 2017.
RCKT Trabodenoson and latanoprost
Glaucoma
 Phase 2
Phase 2 trial failed - noted July 7, 2017.
UTHR Ralinepag ADVANCE OUTCOMES
Pulmonary Arterial Hypertension
 Phase 2
Phase 2 data released July 10, 2017. Primary endpoint met. Secondary missed.
IMRN IMM-124E
Nonalcoholic steatohepatitis (NASH)
 Phase 2
Phase 2 top-line data due 4Q 2017. Interim analysis July 10, 2017 showed no statistical difference with placebo.
PFE Mylotarg (gemtuzumab ozogamicin)
Acute myeloid leukemia (AML)
 -
Advisory Committee Meeting July 11, 2017 voted in favor 6-1 of a favorable risk:benefit profile.
KDMN KD025
Chronic graft-versus-host disease (cGVHD)
 Phase 2
Phase 2 interim data presented July 11, 2017. ORR 71%.
AMGN BLINCYTO
Ph+ R/R ALL
 Approved
PDUFA date under priority review August 14, 2017. Approval announced July 11, 2017.
BMRN Valoctocogene roxaparvovec (BMN 270) - GENEr8-1
Hemophilia A
 Phase 1/2
Phase 1/2 updated data released July 11, 2017. 97% reduction in Annualized Bleed Rate (ABR).
OCUL DEXTENZA
Ocular inflammation and pain following cataract surgery
 CRL
CRL July 25 2016 - due to manufacturing deficiencies. NDA resubmitted, PDUFA date July 19, 2017. Further CRL issued July 11, 2017 with FDA noting deficiencies in manufacturing processes and analytical testing.
SVRA Aironite - INDIE
Heart failure with preserved ejection fraction (HFpEF)
 Phase 2
Phase 2 interim data released July 11, 2017.
AMGN KYPROLIS (ASPIRE)
Relapsed Multiple Myeloma
 Phase 3
Phase 3 trial met primary endpoint. HR 79%.
LGND KYPROLIS (ASPIRE)
Relapsed Multiple Myeloma
 Phase 3
Phase 3 trial met primary endpoint. HR 79%.
NVS Kymriah (CTL019)
Relapsed/Refractory B-Cell Acute lymphoblastic leukemia
 -
Advisory Committee Meeting July 12, 2017 voted 10-0 recommending approval.
JNJ Guselkumab
Plaque psoriasis
 Approved
Approval announced July 13, 2017.
AMGN EVENITY (Romosozumab)
Osteoporosis
 CRL
CRL issued July 16, 2017.
PBYI Neratinib (NERLYNX)
Extended adjuvant HER2-positive early stage breast cancer
 Approved
Approval announced July 17, 2017.
PRTK Omadacycline - oral
Acute bacterial skin and skin structure infections (ABSSSI)
 Phase 3
Phase 3 data released July 17, 2017 - all endpoints met.
CBAY Seladelpar (RESPONSE)
Primary biliary cholangitis (PBC)
 Phase 2
Phase 2 top-line data released late May 2016. Positive interim data from second trial data released July 17, 2017.
GILD Vosevi (Sofosbuvir/Velpatasvir/Voxilaprevir)
Hepatitis C virus (HCV)
 Approved
Approval announced July 18, 2017.
TBPH Revefenacin
COPD
 Phase 3
12 month safety trial data released July 19, 2017 with NDA filing due 4Q 2017.
IRWD IW-3718
Gastroesophageal reflux disease (GERD)
 Phase 2b
Phase 2b data released July 20, 2017. Primary endpoint met but data below expectations.
BVXV M-001
Universal flu vaccine
 Phase 2b
Phase 2b data released July 20, 2017 - endpoints met.
GSK Benlysta (belimumab) - subcutaneous
Systemic lupus erythematosus (SLE)
 Approved
Approval announced July 21, 2017.
GNCA GEN-003
Genital herpes
 Phase 2b
Phase 2b data released January 6, 2017. 12-month data released July 24, 2017. Phase 3 to commence 4Q 2017.
RETA Bardoxolone methyl - CARDINAL
Alport Syndrome
 Phase 2
Phase 2/3 trial initiation announced February 23, 2017. Data from Phase 2 part of trial released July 24, 2017.
MRK KEYNOTE-040 KEYTRUDA
Recurrent or metastatic head and neck squamous cell carcinoma (HNSCC)
 Phase 3
Phase 3 trial did not meet primary endpoint - July 24, 2017. Awarded accelerated approval in 2016.
LJPC Eravacycline (TP-434) - IGNITE4
cIAI (complicated intra-abdominal infections)
 Phase 3
Phase 3 data released July 25, 2017 - primary endpoint met. NDA filing due 1Q 2018.
PCRX EXPAREL
Post surgical pain
 Phase 3
Phase 3 data released July 25, 2017. Upper extremity met primary endpoint, lower extremity trial missed endpoint.
EGRX Ryanodex
Exertional heat stroke (EHS)
 CRL
CRL issued July 26, 2017. Additional trial required.
AZN Durvalumab +/- tremelimumab (MYSTIC)
Lung cancer
Phase 3
Phase 3 data released July 27, 2017 - primary endpoint not met.
DVAX HEPLISAV-B
Hepatitis B
 -
Advisory Committee Meeting July 28, 2017 voted 11 -1 in favor of recommending approval.
NVIV Neuro-Spinal Scaffold: INSPIRE 2.0
Complete Thoracic AIS A Spinal Cord Injury
 Phase 3
Noted July 31, 2017 that enrollment has been temporarily halted as it discusses potential changes to patient enrollment criteria due to patient deaths - enrollment now due to be completed 1H 2018 with Humanitarian Device Exemption (HDE) application due 2H 2018.
RDHL RHB-104 MAP
Crohn’s disease
 Phase 3
Phase 3 DSMB safety analysis released December 13, 2016 - trial to continue as planned. Further analysis July 31, 2017 recommended trial continue as planned.
ICPT Obeticholic acid (OCA) AESOP
Primary Sclerosing Cholangitis (PSC)
 Phase 2
Phase 2 data released July 31, 2017 - primary endpoint met.
ICPT Obeticholic acid (OCA) - CONTROL
NASH patients taking statins
 Phase 2
Phase 2 primary endpoint met - July 31, 2017. One patient death noted but DSMB noted unlikely to be related to OCA (company unsure).
BMY CM-142 – Opdivo
(dMMR) or microsatellite instability high (MSI-H) metastatic colorectal cancer (CRC)
 Approved
Approval announced August 1, 2017.
AGIO IDHIFA (enasidenib) - AG-221
Advanced hematologic malignancies with an IDH2 mutation
 Approved
Approval announced August 1, 2017.
BMY IDHIFA (enasidenib) - AG-221
Advanced hematologic malignancies with an IDH2 mutation
 Approved
Approval announced August 1, 2017.
ACRX Zalviso
Post-operative pain following open abdominal surgery and hip or knee replacement surgery
 Phase 3
Phase 2 data released August 1, 2017.
JNJ Sirukumab
Rheumatoid arthritis
 -
BLA filing announced September 23, 2016. PDUFA estimate September 23, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.
ABBV Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
 Approved
Approval announced August 2, 2017.
TBPH Velusetrag (TD-5108)
Gastroparesis
 Phase 2b
Phase 2b data released August 2, 2017. Primary endpoint not met.
JNJ Imbruvica
Second-line Chronic graft-versus-host disease (GVHD)
 Approved
Approval announced August 2, 2017.
CRIS Fimepinostat (CUDC-907) and venetoclax
Diffuse-large B-cell lymphoma (DLBCL)
 Phase 2
Phase 2 initial data released August 3, 2017 - ORR 19%.
JAZZ Vyxeos
Acute myeloid leukemia
 Approved
Approval announced August 3, 2017.
PFE Xeljanz
Psoriatic arthritis
 -
PDUFA date December 2017. No exact date given. December 31, 2017 noted as last possible date using guidance of "December 31". Advisory Committee Meeting August 3, 2017 voted 10-1 in favor of recommending approval.
ENTA Glecaprevir/Pibrentasvir (G/P)
Hepatitis C virus (HCV)
 Approved
Approval announced August 3, 2017.
ABBV Glecaprevir/Pibrentasvir (G/P)
Hepatitis C virus (HCV)
 Approved
Approval announced August 3, 2017.
LLY Lasmiditan
Migraine
 Phase 3
Phase 3 data released August 4, 2017 - endpoints met. NDA filing due 2H 2018.
BMY Mavacamten (MYK-461) - EXPLORER-HCM
Hypertrophic cardiomyopathy (HCM)
 Phase 2
Phase 2 data released August 7, 2017 - primary endpoint met.
BHC Latanoprostene bunod
Open angle glaucoma (OAG) or ocular hypertension (OHT)
 CRL
CRL issued August 7, 2017.
FGEN Pamrevlumab (FG-3019) - ZEPHYRUS
Idiopathic pulmonary fibrosis
 Phase 2
Phase 2 data released August 7, 2017 - primary endpoint met.
ZYNE ZYN002
Adult Epilepsy Patients with Refractory Focal Seizures
 Phase 2
Phase 2 data released August 7, 2017 - endpoints not met.
ESPR Bempedoic Acid/ Ezetimibe
Hypercholesterolemia
 Phase 2
Phase 2 data released August 8, 2017. Primary endpoint met.
ABIO Gencaro
Chronic Heart Failure
 Phase 2b
Phase 2b interim analysis August 9, 2018 - trial to continue to completion. Data 1Q 2018.
GLPG ISABELA - Ziritaxestat GLPG1690
Idiopathic pulmonary fibrosis
 Phase 2a
Phase 2a top-line data released August 10, 2017. Disease progression halted. Further trials planned.
GILD ISABELA - Ziritaxestat GLPG1690
Idiopathic pulmonary fibrosis
 Phase 2a
Phase 2a top-line data released August 10, 2017. Disease progression halted. Further trials planned.
TGTX Umbralisib (UNITY-NHL)
Follicular lymphoma (FL)
 Phase 2b
Phase 2b enrollment commenced June 2016. First interim analysis August 10, 2017 determined that enrollment in TGR-1202 arm will stop but continue in TG-1101 + TGR-1202 arm. TGR-1202 arm replaced by combo of TG-1101, TGR-1202, and bendamustine.
ISEE Fovista
Wet age-related macular degeneration (Wet-AMD)
 Phase 3
Phase 3 data released December 12, 2016 - primary endpoint not met. Data from third trial released August 14, 2017- primary endpoint not met.
REGN REGN2222
Respiratory syncytial virus (RSV)
 Phase 3
Phase 3 data released August 14, 2017 - primary endpoint not met.
CVM Multikine
Head and Neck cancer
 Phase 3
Initiated Dec 2010. Announced partial clinical hold September 27, 2016. Full clinical hold noted May 2017. Clinical hold released - August 14, 2017.
ZYNE ZYN002 - STOP
Osteoarthritis
 Phase 2
Phase 2 data released August 14, 2017 - primary endpoint not met. However, company still intends to initiate Phase 3 trial pending discussions with FDA.
BMY CM-214 – Opdivo + Yervoy
First-line Renal cell carcinoma (RCC)
 Phase 3
Phase 3 data released August 15, 2017 - one of two co-primary endpoints met.
AZN Lynparza - SOLO 2
Second-line ovarian cancer
 Approved
Approval announced August 17, 2017.
PFE Inotuzumab Ozogamicin
Relapsed or Refractory Acute Lymphoblastic Leukemia in Adults
 Approved
Approval announced August 17, 2017.
IRWD DUZALLO (LESINURAD AND ALLOPURINOL)
Hyperuricemia associated with gout
 Approved
Approval announced August 21, 2017.
RARE Aceneuramic acid extended release (Ace-ER)
GNE Myopathy
 Phase 3
Phase 3 data released August 22, 2017 - endpoints not met.
ADMS GOCOVRI (ADS-5102)
Levodopa-Induced Dyskinesia + Parkinson's disease patients receiving levodopa and experiencing OFF episodes
 Approved
Approval announced August 24, 2017.
NVO Victoza (liraglutide)
Type 2 diabetes
 Approved
Approval announced August 25, 2017.
KMDA KamRAB
Prophylaxis of rabies disease
 Approved
Approval announced August 25, 2017.
XOMA ACZ885 (canakinumab)
CV risk reduction
 Phase 3
Data presented August 27, 2017 showed a 77% reduction in lung cancer mortality.
NVS ACZ885 (canakinumab)
CV risk reduction
 Phase 3
Data presented August 27, 2017 showed a 77% reduction in lung cancer mortality.
AZN Faslodex (fulvestrant)
Monotherapy for expanded use in women with HR+, HER2- advanced breast cancer
 Approved
Expanded approval announced August 28, 2017.
NVS Meropenem-vaborbactam
Complicated urinary tract infections (cUTI)
 Approved
Approved August 29, 2017.
OTIC OTO-104 AVERTS-1
Meniere's disease
 Phase 3
Phase 3 data released August 30 failed to meet primary and key secondary endpoints.
OTIC OTIVIDEX
Ménière's disease
 Phase 3
Phase 3 development suspended following failure of AVERTS-1 trial - August 30, 2017.
NVS Kymriah (CTL019)
Relapsed/Refractory B-Cell Acute lymphoblastic leukemia
 Approved
PDUFA date under priority review announced March 29, 2017. Approval announced early - August 30, 2017.
UTHR Trevyent
Pulmonary Arterial Hypertension (PAH)
 -
Refusal to file Letter (RTF) issued August 31, 2017 in response to NDA filing.
PFE Mylotarg (gemtuzumab ozogamicin)
Acute myeloid leukemia (AML)
 Approved
Approval announced September 1, 2017.
CLLS UCART123 - AMELI-01
Acute myeloid leukemia (AML)
 Phase 1
Clinical hold announced September 4, 2017 following one fatality in the BPDCN clinical trial (ABC study).
NVS Fingolimod
Multiple Sclerosis (MS)
 Phase 3
Phase 3 trial met primary endpoint.
VSTM Duvelisib - DUO
Chronic lymphocytic leukemia (CLL)//Small Lymphocytic Lymphoma (SLL)/Follicular Lymphoma
 Phase 3
Phase 3 data released September 6, 2017 - primary endpoint met.
SRPT Golodirsen - Exon 53
Duchenne muscular dystrophy
 Phase 1/2
Phase 1/2 data released September 6, 2017 - endpoints met.
AMGN Tezepelumab - NAVIGATOR
Asthma
 Phase 2b
Phase 2b data released September 7, 2017 - primary endpoint met.
AZN Tezepelumab - NAVIGATOR
Asthma
 Phase 2b
Phase 2b data released September 7, 2017 - primary endpoint met.
VRNA RPL554
COPD
 Phase 2a
Phase 2a positive data released September 7, 2017.
BMY CM-214 – Opdivo + Yervoy
First-line Renal cell carcinoma (RCC)
 Phase 3
Phase 3 trial stopped early due to clear efficacy.
ABBV Upadacitinib
Atopic Dermatitis
 Phase 2b
Phase 2b data released September 7, 2017 - primary endpoint met.
KURA Tipifarnib - (AIM-HN/SEQ-HN)
Head and neck squamous cell carcinomas (HNSCC)
 Phase 2
Phase 2 trial met primary endpoint prior to completion of enrollment.
SGEN Tisotumab Vedotin (innovaTV 204 )
Cervical Cancer
 Phase 1/2
Phase 1/2 data presented at ESMO 2017. Response rate 32%.
GMAB Tisotumab Vedotin (innovaTV 204 )
Cervical Cancer
 Phase 1/2
Phase 1/2 data presented at ESMO 2017. Response rate 32%.
UTHR Orenitram
Pulmonary arterial hypertension (PAH)
 Phase 3
Phase 3 data due 2H 2018. Independent data monitoring committee (DMC) noted September 8, 2017 that the trial should continue following its interim analysis.
LLY KEYTRUDA + chemo (KEYNOTE-021)
First-Line Nonsquamous Non-small cell lung cancer (NSCLC)
 Phase 2
Phase 2 data presented at ESMO 2017 - ORR 57%, PFS 19 months, HR 0.54
MRK KEYTRUDA + chemo (KEYNOTE-021)
First-Line Nonsquamous Non-small cell lung cancer (NSCLC)
 Phase 2
Phase 2 data presented at ESMO 2017 - ORR 57%, PFS 19 months, HR 0.54
AZN Durvalumab (PACIFIC)
Lung cancer
Phase 3
Late breaker at ESMO September 9, 2017 showed PFS 16.8 months v 5.6 months for placebo.
EXEL Cabozantinib - CABOSUN
First-Line RCC - cancer
 Phase 2
Late breaker at ESMO September 2017 showed PFS 8.6 months versus 5.3 months for sunitinib. HR 0.48
BMY CM-238 – Opdivo (Adjuvant)
Melanoma
 Phase 3
Phase 3 interim data released June 6, 2017 - primary endpoint met. Data presented at ESMO September 10, 2017 - HR 0.65 compared to Yervoy.
MRNS Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)
 Phase 2
Phase 2 top-line data released September 11, 2017.
LGND Ganaxolone - Marigold
CDKL5 Deficiency Disorder (CDD)
 Phase 2
Phase 2 top-line data released September 11, 2017.
REGN Dupixent (dupilumab)
Asthma
 Phase 3
Phase 3 data released September 11, 2017 - primary endpoints met. 46% improvement after 52 weeks in 300 mg group. sBLA filing due by end of 2017.
SNY Dupixent (dupilumab)
Asthma
 Phase 3
Phase 3 data released September 11, 2017 - primary endpoints met. 46% improvement after 52 weeks in 300 mg group. sBLA filing due by end of 2017.
SAGE SAGE-547
SRSE
 Phase 3
Phase 3 data released September 12, 2017 - primary endpoint not met.
ALDX ADX-102 - Reproxalap
Dry eye syndrome
 Phase 2a
Phase 2a data released September 11, 2017 - noted statistically and clinically significant activity.
GSK Shingrix
Shingles
 -
Advisory Committee Meeting September 13, 2017 voted 11-0 in favor. PDUFA estimate October 24, 2017.
VNDA Tradipitant - EPIONE 1
Atopic dermatitis
 Phase 2
Phase 2 data released September 13, 2017 - primary endpoint not met.
AGEN Shingrix
Shingles
 -
Advisory Committee Meeting September 13, 2017 voted 11-0 in favor. Shingrix, containing Agenus' adjuvant, QS-21 Stimulon.
FOLD SD-101
Epidermolysis Bullosa (EB)
 Phase 3
Phase 3 top-line data released September 13, 2017 - endpoints not met.
ABBV MVASITM (bevacizumab-awwb)
Biosimilar candidate to Avastin (bevacizumab)
 Approved
Approved September 14, 2017.
AZN Bydureon
Type 2 Diabetes
Phase 3
Phase 3 Cardiovascular Outcome trial data released September 14, 2017 - primary efficacy objective of a superior reduction in MACE missed statistical significance (p=0.061).
AMGN MVASITM (bevacizumab-awwb)
Biosimilar candidate to Avastin (bevacizumab)
 Approved
Approved September 14, 2017.
PFE XTANDI (PROSPER)
Non-metastatic high risk hormone-sensitive prostate cancer
 Phase 3
Phase 3 data released September 14, 2017 - primary endpoint met.
NEOS NT-0201
Attention deficit hyperactivity disorder (ADHD)
 Approved
Approval announced September 15, 2017.
OPTN XHANCE
Nasal polyps
 Approved
Approval announced September 18, 2017 (prior to IPO).
GSK Trelegy Ellipta (FF/UMEC/VI)
Chronic obstructive pulmonary disease (COPD)
 Approved
Approval announced September 18, 2017.
INVA Trelegy Ellipta (FF/UMEC/VI)
Chronic obstructive pulmonary disease (COPD)
 Approved
Approval announced September 18, 2017.
SUPN SPN-810
Impulsive Aggression in ADHD
 Phase 3
Noted September 18, 2017 that the lower of the two doses will be discontinued following interim analysis.
ABBV Venclexta (MURANO)
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
 Phase 3
Phase 3 data released September 18, 2017 - PFS primary endpoint met.
RHHBY Venclexta (MURANO)
Relapsed or refractory Chronic Lymphocytic Leukemia (CLL)
 Phase 3
Phase 3 data released September 18, 2017 - PFS primary endpoint met.
NBRV Lefamulin - Intravenous and Oral
Moderate to severe Community Acquired Bacterial Pneumonia (CABP)
 Phase 3
Phase 3 data released September 18, 2017 - primary endpoints met.
PFE Sutent (Sunitinib)
Renal cell carcinoma (RCC)
 -
Advisory Committee Meeting September 19, 2017 voted 6-6 the benefits outweigh risks.
KPTI Selinexor - SEAL
Dedifferentiated liposarcoma
 Phase 2/3
Phase 2/3 trial initiated January 2016.Topline data for the Phase 2 portion released September 20, 2017 - PFS HR 0.60. Phase 3 data due 2019.
ALNY Patisiran
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
 Phase 3
Phase 3 top-line data released September 20, 2017 - all endpoints met. NDA filing due by end of 2017.
SNY Patisiran
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
 Phase 3
Phase 3 top-line data released September 20, 2017 - all endpoints met. NDA filing due by end of 2017.
ARAV VRS-317 VELOCITY
Growth hormone deficiency, or GHD - pediatric
 Phase 3
Phase 3 top-line data released September 21, 2017 - primary endpoint not met.
BCRX RAPIVAB (peramivir injection)
Acute uncomplicated influenza to pediatric patients
 Approved
Approved September 21, 2017.
MRK Keytruda
Cancer - Third-line Gastric or Gastroesophageal Junction Adenocarcinoma
 Approved
Approved September 22, 2017 - under priority review.
JNJ Sirukumab
Rheumatoid arthritis
 CRL
CRL issued September 22, 2017. Advisory Committee Meeting August 2, 2017 voted 12-1 against that safety data is adequate to support approval. 13-0 in favor of efficacy.
BMY Opdivo and Yervoy
Previously Treated Hepatocellular Carcinoma
 Approved
Approved under priority review September 22, 2017.
GNCA GEN-003
Genital herpes
 -
Announced September 25, 2017 that development will be ceased.
PRQR QR-010
Cystic Fibrosis
 Phase 1b
Phase 1b top-line data released September 25, 2017. FEV levels not significant across four doses. One of four doses showed significance in subgroup.
PTCT Translarna
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
 -
Received a Refusal to File letter for their NDA Filing - February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. New PDUFA date October 24, 2017. Advisory Committee Meeting September 28, 2017 voted that although it is possible that ataluren may be effective, the data are inconclusive, and more work would be needed to establish whether ataluren is effective.
ZYNE Zygel ZYN002
Fragile X syndrome
 Phase 2
Phase 2 data released September 28, 2017. Company noted data were positive with primary endpoint met. Note though that trial was uncontrolled/open-label.
PRTA PRX003
Psoriatic Arthritis
 Phase 1b
Phase 1b data released September 28, 2017 - insufficient meaningful clinical effect for further development.
ZGNX FINTEPLA (ZX008)
Dravet syndrom
 Phase 3
Phase 3 data released September 29, 2017 - primary endpoint met.
NVO Fiasp (fast-acting insulin aspart)
Type 1 and type 2 diabetes
 Phase 3
Approved September 29, 2017.
MCRB SER-287
Mild-to-Moderate Ulcerative Colitis
 Phase 1b
Phase 1b data released October 2, 2017 - did not perform better than placebo.
ABBV Botox
Forehead lines
 Approved
Approval (third indication) announced October 3, 2017.
RIGL Fostamatinib
Warm Autoimmune hemolytic anemia (WAIHA)
 Phase 2
Phase 2 data from Stage 1 of trial released October 3, 2017 - sufficient efficacy shown to progress to Stage 2.
RDHL BEKINDA
IBS-D
 Phase 2
Phase 2 top-line data released October 3, 2017 - primary endpoint met (borderline) - p=0.05.
LLY Abemaciclib - MONARCH 1 and 2
Second-line hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer and HR+, HER2- breast cancer
 Approved
Approved October 4, 2017.
BLUE Lenti-D - ALD-102
Childhood cerebral adrenoleukodystrophy (CCALD)
 Phase 2/3
Phase 2/3 trial initiated October 2013. Interim top-line data released June 26, 2017. Further data released October 4, 2017 - 88% free of free of major functional disabilities.
CATB Edasalonexent (CAT-1004)
Duchenne muscular dystrophy (DMD)
 Phase 1/2
Phase 1/2 data from open-label extension trial presented October 4, 2017 - deemed sufficient to proceed to Phase 3 development.
VTRS Glatiramer Acetate Injection - Generic for Copaxone
Multiple sclerosis (MS)
 Approved
Approval announced October 4, 2017.
NBIX INGREZZA
Tardive dyskinesia
 Approved
Approval for sNDA 80 mg capsules announced October 5, 2017.
FLXN Zilretta - FX006
Osteoarthritis of the knee
Approved
Approval announced October 6, 2017.
SRPT Golodirsen - Exon 53
Duchenne muscular dystrophy
 Phase 1/2
Phase 1/2 late breaking poster presented at World Muscle Society October 7, 2017.
LLY Abemaciclib - JUNIPER
Thrid line KRAS mutation positive Non-small cell lung cancer (NSCLC)
 Phase 3
Phase 3 data released October 10, 2017 - primary endpoint not met
ANAB ANB020 - etokimab
Moderate-to-severe adult atopic dermatitis
 Phase 2a
Phase 2a positive data released October 10, 2017
VTRS MYL-1401H
Neulasta biosimilar
 CRL
CRL issued October 10, 2017.
ARDX Tenapanor (T3MPO-2)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data released October 11, 2017. Primary endpoint met but with relatively high rates of diarrhea.
ACRX DSUVIA (ARX-04)
Moderate-to-severe acute pain following a surgical procedure
 CRL
CRL issued October 12, 2017.
PFE LYRICA (pregabalin)
Postherpetic neuralgia (PHN)
 Approved
Approval announced October 12, 2017.
ATRS XYOSTED (testosterone enanthate)
Testosterone deficiency
 -
PDUFA date October 20, 2017. Noted October 12, 2017 that the FDA has identified deficiencies with the NDA.
JNJ STELARA (USTEKINUMAB)
Plaque psoriasis
 Approved
Approval announced October 13, 2017.
AERI Rhopressa
Glaucoma
 -
PDUFA date February 28, 2018. Advisory Committee meeting October 13, 2017 voted 9-1 in favor that efficacy outweighs safety risks,
EXEL Cabozantinib (CELESTIAL)
Advanced hepatocellular cancer (HCC)
 Phase 3
Phase 3 data released October 16, 2017 - primary endpoint met. sNDA filing due 1Q 2018.
TAK Cabozantinib (CELESTIAL)
Advanced hepatocellular cancer (HCC)
 Phase 3
Phase 3 data released October 16, 2017 - primary endpoint met. sNDA filing due 1Q 2018.
FENC Pedmark (sodium thiosulfate)
Cisplatin-Induced Hearing Loss
Phase 3
Phase 3 trial met primary endpoint - full data released October 16, 2017.
NVO Semaglutide
Type 2 diabetes
 -
Advisory Committee Meeting October 18, 2017 voted 16-0 in favor.
RHHBY Entrectinib
Solid tumors and ROS1-positive non-small cell lung cancer (NSCLC)
 Phase 2
Updated promising data released October 18, 2017.ORR 69%, PFS 29.6 months.
DRRX POSIMIR
Post-operative pain relief
 Phase 3
CRL Feb 12 2014. Phase 3 commenced November 2015 in response to CRL. Noted October 19, 2017 that primary endpoint was not met.
CRBP Lenabasum (resunab) - DETERMINE
Dermatomyositis
 Phase 2
Phase 2 data released October 19, 2017 - primary endpoint met.
JNJ SIMPONI ARIA
Psoriatic arthritis (PsA)
 Approved
Approval announced October 20, 2017.
JNJ SIMPONI ARIA
Ankylosing Spondylitis
 Approved
Approval announced October 20, 2017.
GSK Shingrix
Shingles
 Approved
Approval announced October 20, 2017.
ATRS XYOSTED (testosterone enanthate)
Testosterone deficiency
 CRL
CRL issued October 20, 2017.
AGEN Shingrix
Shingles
 Approved
Approval announced October 20, 2017.
DBVT Viaskin Peanut
Peanut allergy - ages 4 to 11 years
 Phase 3
Phase 3 top-line data released October 20, 2017 - primary endpoint not met.
ENTA EDP-305 ARGON-2
Non-alcoholic steatohepatitis (NASH)
 Phase 1
Phase 1 data at AASLD meeting - October 23, 2017.
ALXN Eculizumab
Refractory generalized myasthenia gravis (gMG)
 Approved
Approval announced October 23, 2017.
GBT GBT440
Hypoxemia in patients with idiopathic pulmonary fibrosis (IPF) who are on supplemental oxygen at rest
 Phase 2a
Phase 2a discontinuation announced October 23, 2017 due to lack of efficacy.
PTCT Translarna
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
 CRL