Historical Catalyst Calendar

Biotech Stock Historical Catalysts listed from 2009.

What's included?

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

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

August 2016 ~ Small/mid cap Phase 2 and Phase 3 data releases.

January 2016 ~ Large cap releases added (data and regulatory).

Note: Most bankrupt or acquired companies are not included.

Ticker Drug Stage Catalyst
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.
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.
INCY Epacadostat with Keytruda - ECHO-202/KEYNOTE-037
NSCLC, RCC, Triple-Negative Breast, Ovarian, Urothelial Carcinoma, Solid Tumors
-
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.
ZFGN ZGN-1061
Diabetes
Phase 1
Phase 1 positive safety data released May 4, 2017. Phase 2 initiation due 2H 2017.
ACHN JNJ-4178
Hepatitis C (HCV)
Phase 2a
Phase 2a ongoing. Updated data presented at EASL April 22, 2017.
MRK Januvia (Sitagliptin)
Type 2 Diabetes
CRL
CRL issued April 7 2017. sNDA for approved drug requested to include data on cardiovascular effects
BMY CM - 067 Opdivo in combination with Yervoy
Melanoma
Phase 3
Phase 3 first overall survival (OS) positive data released April 3, 2017
AZN Tagrisso
Epidermal growth factor receptor (EGFR) T790M mutation-positive non-small cell lung cancer (NSCLC)
Approved
Approval announced March 31, 2017.
MTFB Iclaprim -REVIVE-1
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 data released April 18, 2017 - primary endpoint of non-inferiority met.
AUPH Voclosporin - AURION trial
Lupus
Phase 2
48-week data from open-label AURION study presented March 27, 2017 - primary endpoint met.
AGN JUVÉDERM VOLLURE
Correction of moderate to severe facial wrinkles and folds
Approved
Approval announced March 20, 2017.
AKTX Coversin
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.
GALT GR-MD-02
Plaque psoriasis
Phase 2
Phase 2 data released March 6, 2017.
MRK Odactra (MK-8237)
House dust mite allergies
Approved
Approved March 1, 2017.
PFE Avelumab
Urothelial Carcinoma
Approved
Approval announced May 9, 2017.
DEPO GRALISE
Postherpetic neuralgia - shingles
Approved
Approved January 28, 2011.
JAZZ JZP-6
Fibromyalgia
CRL
CRL received October 11, 2010.
ARLZ VIMOVO
Gastric ulcers
Approved
Approved April 30, 2010.
SGEN ADCETRIS
Hodgkin lymphoma and Anaplastic large cell lymphoma
Approved
Approval announced August 19, 2011.
PCRX EXPAREL
Postsurgical pain
Approved
Approved October 31, 2011.
INCY Jakafi
Myelofibrosis
Approved
Approved November 16, 2011.
ATRS Anturol
Overactive bladder
Approved
Approved December 8, 2011.
INSY SUBSYS
Cancer pain
Approved
Approved January 5, 2012.
ALKS BYDUREON
Type 2 diabetes
Approved
Approved January 27, 2010.
ALKS BYDUREON
Type 2 diabetes
CRL
CRL received March 15, 2010.
FCSC Laviv
Wrinkles
CRL
CRL issued December 18, 2009.
FCSC Laviv
Wrinkles
Approved
Approved June 22, 2011.
PRTK Intermezzo
Insomnia
CRL
CRL issued November 2, 2009.
PRTK Intermezzo
Insomnia
Approved
Approved November 23, 2011.
GTXI Toremifene
Reduce fractures in men with prostate cancer
CRL
CRL issued November 2, 2009.
SPPI FUSILEV
Colorectal cancer
CRL
CRL received October 9, 2009.
SPPI FUSILEV
Colorectal cancer
Approved
Approved April 29, 2011.
ZGNX Sumavel
Migraine
Approved
Approved July 16, 2009.
ARDM Sumavel
Migraine
Approved
Approval granted July 16, 2009.
SPPI ZEVALIN
Non-Hodgkin’s lymphoma
CRL
CRL received July 5, 2009.
SPPI ZEVALIN
Non-Hodgkin’s lymphoma
Approved
Approved September 4, 2009.
CRIS Vismodegib
Basal Cell Carcinoma
Approved
Approved January 30, 2012.
CORT Corlux - Korlym
Cushing's Syndrome
PDUFA
Approved February 17, 2012.
WINT Surfaxin
Respiratory Distress Syndrome
Approved
Approved March 6, 2012 - under Discovery Laboratories, Inc.
PLX Taliglucerase alfa
Gaucher disease
CRL
CRL issued February 25, 2011.
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.
VVUS Qnexa
Obesity
CRL
October 28, 2010.
VVUS Qnexa
Obesity
Approved
Approved July 17, 2012.
ARNA Lorcaserin
Obesity
Approved
Approved June 27, 2012.
SUPN Oxtellar
Epilepsy
Approved
Approved October 22, 2012.
EXEL Cabozantinib
Medullary thyroid cancer
Approved
Approved November 29, 2012.
NAVB Lymphoseek
Lymphatic-tissue tracing agent
CRL
CRL issued September 10, 2012.
NAVB Lymphoseek
Lymphatic-tissue tracing agent
Approved
Approved March 13, 2013.
AVDL Bloxiverz (neostigmine methylsulfate)
Reversal of the effects of non-depolarizing neuromuscular blocking agents after surgery
Approved
Approved June 3, 2013.
TBPH VIBATIV (telavancin hydrochloride) for injection
Hospital-acquired and ventilator-associated bacterial pneumonia (HABP/VABP)
Approved
Approved June 21, 2013.
ABBV Ibrutinib
Relapsed or refractory MCL mantle cell lymphoma
Approved
Approved November 13, 2013.
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.
ABBV Imbruvica
Deletion 17p
Approved
Approved July 19, 2014.
ABBV VIEKIRA PAK
HCV - genotype 1
Approved
Approved December 19, 2014.
ABBV IMBRUVICA
Waldenström’s Macroglobulinemia
Approved
Approved January 29, 2015 - PCYC
AMGN Corlanor
Chronic Heart Failure
Approved
Approved April 15, 2015.
TBPH BREO ELLIPTA
Asthma
Approved
Approved April 30, 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
XBIT MABp1
Hidradenitis Suppurativa (HS)
Phase 2
Phase 2 data released February 16, 2017.
AGN Botox
Depression
Phase 2
Phase 2 top-line data released April 5, 2017 - primary endpoint missed. Phase 3 planned.
AGN Sarecycline
Severe acne vulgaris
Phase 3
Phase 3 data released March 27 2017 - primary endpoints met. NDA filing due 2H 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.
PBYI Roche (RHHBY) - Perjeta + Herceptin (APHINITY)
Adjuvant HER2+ breast cancer
Phase 3
Note this trial is conducted by competitor Roche (RHHBY), NOT by Puma. The event was likely to have a significant impact on the share price of Puma (e.g. positive data would likely have a negative impact on Puma and vice versa). Data were released March 2, 2017 - primary endpoint was met.
MRK KEYTRUDA
First and Second line locally advanced or metastatic urothelial cancer - bladder cancer
Approved
Approved May 18, 2017.
GEMP Gemcabene - COBALT-1
Homozygous Familial Hypercholesterolemia (HoFH)
Phase 2b
Phase 2b interim data released January 30, 2017.
PFE PF-06425090
C. difficile Infection
Phase 2
Phase 2 positive data released January 26, 2017.
VRX Brodalumab
Psoriasis
Approved
Approved February 15, 2017.
SNY Dupixent (dupilumab)
Eosinophilic esophagitis
Phase 2
Phase 2 data released May 4, 2017.
SNY Dupixent (dupilumab)
Atopic dermatitis
Approved
Approved under priority review - March 28, 2017.
SNY Sarilumab
Rheumatoid arthritis
Approved
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
REGN Dupixent (dupilumab)
Eosinophilic esophagitis
Phase 2
Phase 2 data released May 4, 2017.
REGN Dupixent (dupilumab)
Atopic dermatitis
Approved
Approved under priority review - March 28, 2017.
REGN Sarilumab
Rheumatoid arthritis
Approved
CRL October 28, 2016. Resubmitted with FDA Approval announced May 22, 2017.
PFE hGH-CTP (MOD-4023)
Adults - growth hormone deficiency
Phase 3
Phase 3 data released December 30, 2016 - primary endpoint not met.
PFE Avelumab
Metastatic Merkel cell carcinoma
Approved
Priority review announced November 29, 2016. Approval announced March 23, 2017.
PFE IBRANCE (palbociclib)
HR+, HER2- Metastatic Breast Cancer
Approved
sNDA acceptance announced December 21, 2016. Approval announced March 31, 2017.
NVS RELAX-AHF-2 (RLX030)
Acute heart failure
Phase 3
Phase 3 data released March 22, 2017 - primary endpoints not met.
NVS Rydapt (Midostaurin - PKC412)
Acute myeloid leukemia (AML)
Approved
Priority review granted November 14, 2016. Approval announced April 28, 2017.
NVS LEE011
HR+/HER2- advanced breast cancer
Approved
Priority review granted November 1, 2016. Approval announced March 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.
MRK Keytruda
First-Line Treatment of Metastatic Non-Squamous Non-Small Cell Lung Cancer
Approved
Approved May 10, 2017.
MRK KEYTRUDA
Relapsed or Refractory Classical Hodgkin Lymphoma
Approved
Approval announced March 14, 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.
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.
LLY Abemaciclib - MONARCH 2
Second-line hormone-receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2-) breast cancer
Phase 3
Phase 3 data released March 20, 2017 - PFS primary endpoint met.
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.
JNJ Imetelstat - IMbarkStudy
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.
JAZZ Xyrem
Pediatric narcolepsy patients with cataplexy
Phase 2/3
Phase 2/3 positive data released April 24, 2017. sNDA filing due 4Q 2017.
JAZZ JZP-110
Excessive sleepiness associated with obstructive sleep apnea
Phase 3
Phase 3 data released March 20, 2017 - primary endpoints met.
JAZZ JZP-110
Excessive sleepiness associated with narcolepsy
Phase 3
Phase 3 data released April 26, 2017. NDA to be filed late 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.
CELG Ozanimod - SUNBEAM
Relapsing Multiple Sclerosis
Phase 3
Phase 3 data released February 17, 2017 - primary endpoint met.
CELG Ozanimod - RADIANCE
Relapsing Multiple Sclerosis
Phase 3
Phase 3 data due released May 22, 2017 - primary endpoint met, disability endpoint not met.
CELG Demcizumab plus carboplatin and pemetrexed - DENALI
First-line non-squamous Non-small cell lung cancer (NSCLC)
Phase 2
Announced November 1, 2016 that it has discontinued further enrollment. Phase 2 data released May 8, 2017 - endpoints not met.
CELG Demcizumab plus Abraxane - YOSEMITE
First-line pancreatic cancer
Phase 2
Phase 2 data released April 10, 2017 - primary endpoint not met.
CELG REVLIMID
Multiple myeloma (MM) - autologous stem cell transplant (ASCT)
Approved
Approved February 22, 2017.
BMY CM-143 – Opdivo
Second-line Glioblastoma (GBM) cancer
Phase 3
Phase 3 data released April 3, 2017 - primary endpoint not met.
AZN Tralokinumab (STRATOS1)
Severe, uncontrolled asthma
Phase 3
Phase 3 trial did not meet primary endpoint - May 10, 2017.
AZN Lynparza - SOLO 2
Second-line ovarian cancer
Phase 3
Data presented March 14, 2017 showed significant PFS benefit.
AZN Lynparza
Breast cancer
Phase 3
Phase 3 data released February 17, 2016 - primary endpoint 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.
AZN Urothelial carcinoma - Bladder cancer
Bladder cancer
Approved
BLA acceptance announced December 9, 2017. PDUFA under priority review. Approval announced May 1, 2017.
AZN ZS-9
Hyperkalaemia
CRL
CRL issued May 26, 2016.
AZN ZS-9
Hyperkalaemia
CRL
Second CRL issued March 17, 2017.
AZN Saxagliptin and dapagliflozin
Type-2 diabetes
Approved
Approval announced February 28, 2017.
AMGN Repatha
Cardiovascular disease
Phase 3
Phase 3 cardiovascular outcomes study data released March 17, 2017 - endpoints met but down on expectations.
AMGN Parsabiv
Secondary hyperparathyroidism (SHPT)
Approved
Approved February 7, 2017.
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.
AGN ESMYA (Venus II)
Uterine Fibroids
Phase 3
Phase 3 data released January 18, 2017 - endpoints met.
AGN AVYCAZ (ceftazidime and avibactam)
Complicated Urinary Tract Infections (cUTI)
Approved
sNDA approval announced January 30, 2017.
AGN Oxymetazoline HCl cream 1.0%
Facial Erythema (Redness) Associated with Rosacea
Approved
Approved January 19, 2017.
AGN Linaclotide 72 mcg dose for use in the treatment of adults
Chronic idiopathic constipation (CIC)
Approved
sNDA approval announced January 26, 2017.
ABBV ABT-494
Crohn’s Disease
Phase 2
Phase 2 positive data at DDW Meeting May 9, 2017. Phase 3 trial to be initiated later in 2017.
ABBV Imbruvica
Marginal zone lymphoma
Approved
sNDA filing under priority review approved January 19, 2017.
NTRP Bryostatin
Alzheimer's disease
Phase 3
Phase 2 data released May 1, 2017 - primary endpoint not met.
CTIC PIXUVRI
B-cell non-Hodgkin lymphoma
CRL
CRL received April 9, 2010.
ACOR Ampyra
Improve walking in patients with multiple sclerosis
Approved
Approved January 22, 2010.
ACOR Ampyra
Improve walking in patients with multiple sclerosis
-
Decision on Inter Partes Review (IPR) released March 10, 2017 - patents upheld.
AMPH Naloxone Intranasal
Opioid overdose
CRL
CRL announced February 21, 2017.
AMPH Primatene Mist (epinephrine inhalation aerosol)
Asthma
CRL
CRL issued December 27, 2016.
NDRM ND0612H (Trial 006)
Severe Parkinson's Disease
Phase 2
Phase 2 trial met key primary and secondary endpoints - March 1, 2017.
LXRX Sotagliflozin
Type 1 Diabetes
Phase 2
Phase 2 trial did not meet primary endpoint.
SCYX Intravenous SCY-078
Phase 1
Clinical hold placed on the initiation of intravenous (IV) SCY-078 until the FDA completes a review of all data. Clinical hold follows three mild-to-moderate thrombotic events in healthy volunteers - announced March 2, 2017.
XBIT 514G3
Staphylococcus Aureus Bacteremia
Phase 1/2
Phase 1/2 top-line data released April 3, 2017. 4 patient deaths noted.
VRTX Tezacaftor (VX-661) / 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.
HALO FOLFIRINOX and PEGPH20
Pancreatic and gastric cancers
Phase 1/2
Phase 1/2 enrollment closed due to lack of efficacy.
TENX Levosimendan
Septic shock
Phase 2b
Phase 2b trial did not meet primary endpoint.
NOVN SB206
Human papillomavirus (HPV)
Phase 2
Phase 2 data released November 29, 2016. Primary endpoint met.
NOVN SB208
Fungal infections
Phase 2
Phase 2 data released April 12, 2017. Primary endpoint met.
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.
VRTX KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
Approved
Approved December 29, 2014.
SCMP Amitiza/Lubiprostone
Opioid-induced bowel dysfunction
Approved
Approved April 23, 2013.
IMGN Kadcyla
2nd-line HER2+ Metastatic Breast Cancer
Approved
Approved February 22, 2013 - under priority review.
HEB Ampligen
CFS
CRL
CRL issued December 1, 2009.
HEB Ampligen
CFS
CRL
CRL February 4, 2013.
CPRX Firdapse
MuSK-antibody positive myasthenia gravis (MuSK-MG)
Phase 2
Phase 2 top-line data met endpoints - March 15, 2017. Pivotal trial planned.
MDCO Inclisiran
Hypercholesterolemia
Phase 2
Phase 2 Day 180 data presented at AHA Scientific Sessions on November 15, 2016.
VRTX Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - One Copy of the F508del Mutation and a Second Mutation that Results in Residual CFTR Function
Phase 3
Phase 3 data released March 28, 2017 - primary endpoint met. NDA filing due 3Q 2017.
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.
LPTX CureXcell
Diabetic Foot Ulcers
Phase 3
Phase 3 trial did not meet primary endpoint.
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.
CHMA Mycapssa
Acromegaly
CRL
CRL April 15, 2016.
TGTX TG-1101
Multiple Sclerosis
Phase 2
Phase 2 preliminary results presented at American Academy of Neurology (AAN) annual meeting April 28, 2017.
SAGE SAGE-217
Major Depressive Disorder (MDD)
Phase 2
Phase 2 top-line data from open-label trial released February 13, 2017
SAGE SAGE-217
Parkinson’s disease (PD)
Phase 2
Phase 2 initiation announced December 13, 2016. Data from Part A data released May 9, 2017. Part B data due 2H 2017.
OPK hGH-CTP
Adults - growth hormone deficiency
Phase 3
Phase 3 data released December 30, 2016 - primary endpoint not met.
VICL ASP0113 Vaccine
Kidney transplant recipients
Phase 2
Phase 2 data released September 19, 2016 - did not meet primary endpoint.
LIFE Resolaris
Limb girdle muscular dystrophy (LGMD) 2B / Adult patients with facioscapulohumeral muscular dystrophy (FSHD)
Phase 1/2
Phase 1b/2 data released December 13, 2016.
ZSAN M207
Migraine
Phase 3
Phase 3 trial data due released February 13, 2017 - primary endpoints met.
TTPH Eravacycline (TP-434) - IGNITE3
cUTI - complicated urinary tract infections
Phase 3
Phase 3 trial did not meet endpoints September 8, 2015.
ICPT Obeticholic acid (OCA)
Primary biliary cirrhosis (PBC) - POISE
Approved
Approved May 27 2016.
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.
CORI Corplex Donepezil
Alzheimer's disease
-
Pilot bioequivalence data due released May 11, 2017. Pivotal trial to commence fall of 2017 with preliminary data due February 2018.
NVCR Tumor Treating Fields (TTFields) - INNOVATE
Ovarian cancer
Phase 2
Phase 2 data presented December 12, 2016. Further promising PFS data released March 31, 2017.
NLNK Indoximod in combination with pembrolizumab
Melanoma - cancer
Phase 2
Phase 2 interim analysis released at AACR meeting April 4, 2017.
OCUL DEXTENZA
Allergic conjunctivitis
Phase 3
Phase 3 trial did not meet primary endpoint - June 6, 2016.
IRWD Linaclotide 72 mcg dose for use in the treatment of adults
Chronic idiopathic constipation (CIC)
Approved
sNDA approval announced January 26, 2017.
PRTK Omadacycline
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 top-line data met endpoints - June 16, 2016
LXRX Sotagliflozin
Type 1 Diabetes
Phase 3
Phase 3 data from second trial released December 21, 2016 - primary endpoint met.
AMRN Vascepa REDUCE-IT outcomes trial
High Triglycerides With Mixed Dyslipidemia
Approved
Approved July 26, 2012.
KURA Tipifarnib
HRAS mutant solid tumors - cancer
Phase 2
Phase 2 ongoing data released March 6, 2017 - further data due 2H 2017.
XOMA XOMA 213
Hyperprolactinemia
Phase 2
Phase 2 proof-of-concept results released April 24, 2017.
PTIE Remoxy
Chronic pain
CRL
CRL received June 24, 2011.
PTIE Remoxy
Chronic pain
CRL
CRL received September 26, 2016.
TXMD Yuvvexy
Moderate-to-severe vaginal pain
CRL
CRL announced May 8, 2017. Company noted that FDA cited a lack of long-term endometrial safety data.
KITE Axicabtagene ciloleucel (KTE-C19) - ZUMA-1
Refractory Non-Hodgkin's Lymphoma (NHL) - cancer
Phase 2
Phase 2 pivotal top-line data released September 26, 2016. Primary analysis due 1Q 2017. BLA filing due late 2016.
KITE Axicabtagene ciloleucel (KTE-C19) - ZUMA-1
Refractory Non-Hodgkin's Lymphoma (NHL) - cancer
Phase 2
Phase 2 primary analysis released February 28, 2017 showed trial met primary endpoint. 31% CR for DLBCL.
EIGR Lonafarnib - LOWR HDV
Chronic hepatitis delta virus (HDV)
Phase 2
Phase 2 end of trial data released April 21, 2017. Meeting with FDA scheduled for 4Q 2017.
NVLS N91115
Cystic Fibrosis - two copies of the F508del mutation
Phase 2
Phase 2 data released November 28, 2016. Primary endpoint not met.
LXRX Telotristat etiprate (XERMELO)
Carcinoid Syndrome
Approved
Approved February 28, 2017.
AAAP Lutathera
Inoperable progressive midgut NETs
CRL
PDUFA date under priority review was December 28, 2016. CRL issued December 21, 2016.
TSRO Rolapitant - intravenous (IV)
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
CRL
CRL issued January 11 2017.
SGYP Trulance (plecanatide)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data from first trial met primary endpoint - December 9, 2016.
SGYP Trulance (plecanatide)
Constipation-predominant irritable bowel syndrome (IBS-C)
Phase 3
Phase 3 data from second trial met primary endpoints - December 9 and 22, 2016.
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.
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.
CEMP Solithera - intravenous
Community-acquired bacterial pneumonia (CABP)
CRL
CRL announced December 29, 2016. Further trials required.
ATRS OTREXUP
Rheumatoid arthritis (RA)
Approved
Approved Oct 14, 2013.
AUPH Voclosporin - AURORA
Lupus
Phase 2b
Phase 2b data released August 2016. Primary endpoint met but with high number of deaths.
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.
AVEO TIVO-3 - tivozanib
Third line treatment of patients with renal cell cancer
CRL
CRL issued June 10, 2013.
AVIR BTA585
Respiratory syncytial virus (RSV)
Phase 2a
Phase 2a data released February 1, 2017 - primary endpoint not met.
AVIR Vapendavir - SPIRITUS trial
Human rhinovirus (HRV)
Phase 2b
Phase 2b top-line data released February 13, 2017 - primary endpoint not met.
AXON Nelotanserin
Visual hallucinations in subjects with Lewy body dementia
Phase 2
Phase 2 preliminary data released February 13, 2017. Primary endpoint hit, secondary endpoints missed. Full study to be completed mid-2017. Phase 3 trial to be initiated 2H 2017.
BCRX BCX7353 - APeX-1
Reduce or eliminate attacks in HAE patients
Phase 2
Phase 2 interim data released February 27, 2017.
BCRX Peramivir - Study 303
INFLUENZA ACUTE
Approved
Approved December 22, 2014.
BDSI Clonidine gel
Painful diabetic neuropathy (PDN)
Phase 2b
Phase 2b data released December 13, 2016 - primary endpoints not met.
BDSI BEMA Buprenorphine/Naloxone (BNX) - BUNAVAIL
Opioid dependence
Approved
Approved June 6, 2014.
BLPH INOpulse delivery device
Pulmonary Hypertension associated with Idiopathic Pulmonary Fibrosis (PH-IPF)
Phase 2
Phase 2 initiated May 2016. Data presented at American Thoracic Society (ATS) Conference, May 21 2017. Phase 2b planned.
BMRN Vimizim (GALNS)
(MPS IVA) Morquio A Syndrome
Approved
Approved February 14, 2014.
BPMX BPX-01
Acne
Phase 2b
Phase 2b data released May 3, 2017 - primary endpoint met, secondary missed.
BTX OpRegen
Dry age-related macular degeneration (AMD)
Phase 2
Phase 2 second cohort underway. Interim data presented at May 8, 2017 at ARVO conference.
CAPR CAP-1002 HOPE-Duchenne
Duchenne Muscular Dystrophy (DMD)
Phase 1/2
Phase 1/2 data released April 25, 2017. Noted improvement in certain measures of cardiac and upper limb function. 12-month data due 4Q 2017.
CAPR CAP-1002 ALLSTAR
Myocardial infarction (heart attack)
Phase 2
Phase 2 data released May 12, 2017 - primary endpoint unlikely to be met.
CATB CAT-1004
Duchenne muscular dystrophy (DMD)
Phase 1/2
Phase 1/2 top-line data released January 31 - primary endpoint not met.
CDTX CD101 Topical - RADIANT
Vulvovaginal candidiasis (VVC)
Phase 2
Phase 2 data released February 21, 2017 - efficacy not evident, Development to be discontinued.
CEMP Solithromycin
NASH
Phase 2
Phase 2 interim data released September 30, 2016.
CEMP Solithromycin
NASH
Phase 2
Noted February 28, 2017 that development has been suspended due to lack of efficacy.
CEMP Taksta - Fusidic acid
Acute bacterial skin and skin structure infections (ABSSSI)
Phase 3
Phase 3 data released February 24, 2017 - endpoints met.
CERC CERC-301
Major depressive disorder (MDD)
Phase 2
Phase 2 top-line data released November 29, 2016 failed to meet primary endpoint.
CHRS CHS-1420
Psoriasis
Phase 3
Phase 3 top-line data met endpoints August 2016. Full data due released January 10, 2016.
CLVS Rociletinib
Mutant EGFR T790M-positive lung cancer
CRL
CRL June 28, 2016. Phase 3 TIGER-3 also terminated.
CMRX Brincidofovir - Advise trial
Adenovirus Infection
Phase 3
Phase 3 interim analysis released May 2016. Final data released February 22, 2017.
CRBP Anabasum (Resunab)
Cystic Fibrosis
Phase 2
Phase 2 data released March 30, 2017 - primary endpoint met but some concerns regarding efficacy.
CYTR Aldoxorubicin with doxorubicin
Soft tissue sarcomas refractory cancer
Phase 3
Phase 3 PFS data released July 2016 showed no significant improvement in PFS. Second analysis released November 29, 2016 showed improvement in population with leiomyosarcoma and liposarcoma.
DCTH CHEMOSAT system
Unresectable metastatic ocular melanoma in the liver
CRL
CRL September 13, 2013.
DEPO SEFELSA
Hot flashes
CRL
CRL issued May 31, 2013.
DERM Cimzia
Moderate-to-severe plaque psoriasis
Phase 3
Phase 3 data from first trial released October 2016 - endpoints met. Second trial data released December 8, 2016 - endpoints met. Data from third and final trial met primary endpoint - January 19, 2017.
DMTX DTX101
hemophilia B
Phase 1/2
Phase 1/2 initial data released January 31, 2017.
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
DVAX HEPLISAV-B
Hepatitis B
CRL
CRL issued February 25, 2013.
DVAX HEPLISAV-B
Hepatitis B
CRL
CRL November 14, 2016
EBIO Isunakinra (EBI-005)
Moderate to severe dry eye disease
Phase 3
Phase 3 top-line data showed endpoints not met May 18, 2015.
EBS BioThrax
Anthrax Vaccine Adsorbed
Approved
Approved November 24, 2015.
EBS Anthrax Immune Globulin Intravenous (Human) [AIGIV]
Anthrax
PDUFA
Approved March 25, 2015.
Polymyxin B Hemoperfusion (PMX)
Endotoxemic septic shock
Phase 3
Phase 3 trial failed to meet endpoints.
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.
EGRX Docetaxel Injection
Breast, non-small cell lung, prostate gastric adenocarcinoma, and head and neck cancer
Approved
Approved December 24, 2015.
EGRX Ryanodex
Malignant hyperthermia - cancer
Approved
Approved July 23, 2014 under priority review.
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.
ENDP AVEED (testosterone undecanoate) intramuscular injection
Men diagnosed with hypogonadism
CRL
CRL issued September 5, 2013.
ENDP AVEED (testosterone undecanoate) intramuscular injection
Men diagnosed with hypogonadism
Approved
Approved March 6, 2014.
ENTA VIEKIRA PAK
HCV - genotype 1
Approved
Approved December 19, 2014.
EXEL CABOMETYX (cabozantinib)
Metastatic renal cell cancer (RCC)
Approved
Approved April 25, 2016.
EXEL Vemurafenib
BRAF V600 Mutation-Positive Advanced Melanoma - Cancer
Approved
Approved November 10, 2015.
FGEN Roxadustat FG-4592
Anemia in chronic kidney disease
Phase 3
Phase 3 data from China trial released January 30, 2017. Primary endpoints met.
AVDL Akovaz
Hypotension
Approved
Approved May 2, 2016.
AVDL VAZCULEP (phenylephrine hydrochloride)
Clinically important hypotension resulting primarily from vasodilation in the setting of anesthesia
Approved
Approved June 30, 2014.
FOLD ATB200
Pompe Disease
Phase 1/2
Phase 1/2 additional data released February 15, 2017. Further data released May 15, 2017.
FOMX FMX101
Acne
Phase 3
Phase 3 data released March 27, 2017 - three of four primary endpoints met across two trials.
GALE NeuVax (E75) - PRESENT
Low-to-intermediate HER2+ breast cancer
Phase 3
Phase 3 trial terminated due to poor efficacy.
GERN Imetelstat - IMbarkStudy
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.
GNCA GEN-003
Genital herpes
Phase 2b
Phase 2b virologic efficacy data released September 2016.
GNCA GEN-003
Genital herpes
Phase 2b
Phase 2b data released January 5, 2017.
GWPH Sativex
Cancer pain
Phase 3
Phase 3 trials did not meet endpoints.
HALO PEGPH20
Pancreatic cancer
Phase 2
Primary endpoint met.
HALO HYQVIA
Subcutaneous treatment for patients with primary immunodeficiency (PI)
CRL
CRL issued August 1, 2012.
HALO HYQVIA
Subcutaneous treatment for patients with primary immunodeficiency (PI)
Approved
Approved September 12, 2014.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
CRL
CRL issued March 19, 2010.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
CRL
CRL March, 28 2013.
HRTX APF530 - Sustol
Prevention of acute- and delayed-onset chemotherapy-induced nausea and vomiting (CINV)
Approved
CRL Mar 28 2013. Approved August 10, 2016.
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
IMGN Kadcyla GATSBY trial
Cancer - metastatic HER2+ gastric cancer
Phase 3
Phase 3 endpoint not met - October 22, 2015.
INCY Jakafi (ruxolitinib) (RESPONSE)
Polycythemia Vera
Approved
Approved December 4, 2014.
INNL Cogenzia
Diabetic foot ulcer infections
Phase 3
Phase 3 data did not meet endpoints - November 3, 2016.
INSY Dronabinol Oral Solution
AIDS-related anorexia
Approved
Announced approval July 5, 2016.
IONS IONIS-TTRRx
NEURO-TTR - familial amyloid polyneuropathy (FAP).
Phase 3
Phase 3 data released May 15, 2017 - primary endpoint met but with safety concerns.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Phase 3
Phase 3 ENDEAR trial met primary endpoint August 1, 2016.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Phase 3
Phase 3 CHERISH data met primary endpoint - November 7, 2016.
IONS SPINRAZA - Nusinersen
Spinal muscular atrophy (SMA)
Approved
Approved December 23, 2016.
IONS Volanesorsen - APPROACH
Familial chylomicronemia syndrome (FCS)
Phase 3
Phase 3 data released March 6, 2017 - primary endpoint met. Relative safety concerns noted.
IPXL IPX066
Parkinson's
CRL
CRL issued January 21, 2013.
IPXL IPX066
Parkinson's
Approved
Approved January 8, 2015.
IRWD Linaclotide Colonic Release
Irritable Bowel Syndrome with Constipation (IBS-C)
Phase 2b
Phase 2b data released December 22, 2016.
ITCI ITI-007-302
Schizophrenia
Phase 3
Phase 3 trial failed - September 28, 2016.
KERX Auryxia
Hyperphosphatemia
Approved
Approved Sept 5, 2014.
KMDA Inhaled formulation of AAT
AATD - Alpha-1 Antitrypsin deficiency
Phase 2
Top-line data released August 30, 2016. Primary endpoint met
KPTI Selinexor - STORM
Quadruple Refractory Multiple Myeloma
Phase 2
Phase 2 positive top-line data released September 6, 2016
KPTI Selinexor - SADAL
Cancer - 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.
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.
LJPC LJPC-501
Catecholamine-resistant hypotension (CRH)
Phase 3
Phase 3 data released February 27, 2016 - primary endpoint hit. Secondary endpoints not mentioned. NDA filing due 2H 2017.
LJPC LJPC-501
Catecholamine-resistant hypotension (CRH)
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.
LPCN LPCN 1021
Men with low testosterone (Low T)
CRL
CRL issued June 29, 2016.
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.
MACK ONIVYDE
Cancer - second line pancreatic
Approved
Approved October 22, 2015.
MCRB SER-109
Recurrent C. Difficile infection
Phase 2
Phase 2 interim data did not meet primary endpoint.
MDCO CARBAVANCE
Complicated urinary tract infections (cUTI)
Phase 3
Phase 3 data met endpoints June 27, 2016.
MDCO 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.
MDCO RAPLIXA
Hemostasis
Approved
Approved April 30, 2015.
MDCO IONSYS
Acute postoperative pain
Approved
Approved April 30, 2015.
MDCO Oritavancin
ABSSSI
Approved
Approved August 6, 2014 under priority review
MESO MPC-150-IM
Chronic heart failure (CHF)
Phase 3
Noted April 10, 2017 that interim analysis has been completed - trial to continue as planned.
MNKD AFREZZA
Type 1/2 diabetes
CRL
CRL issued March 15, 2010.
MNKD AFREZZA
Type 1/2 diabetes
CRL
CRL received January 19, 2011.
MNKD AFREZZA
Type 1/2 diabetes
Approved
Approved June 27, 2014.
MNTA Generic Copaxone
Multiple sclerosis
Approved
Approved April 16 2015.
MRNS Ganaxolone
Focal onset seizures
Phase 3
Phase 3 data did not meet endpoints - June 13, 2016
NAVB Lymphoseek
Head and Neck Cancer
Approved
Approved June 13, 2014.
NBIX INGREZZA
Tourette syndrome - adults
Phase 2
Phase 2 data released January 18, 2017 did not meet primary endpoint.
NEOS Adzenys XR-ODT
Attention deficit hyperactivity disorder (ADHD)
Approved
Approved January 27, 2016.
NEOT LIPO-202
Reduction of central abdominal bulging
Phase 3
Phase 3 endpoints not met December 14, 2015.
NKTR NKTR-181
Lower back pain
Phase 3
Phase 3 data released March 20, 2017 - endpoints met.
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.
NKTR ADYNOVATE
Hemophilia A
Approved
Approved November 16, 2015.
NKTR MOVANTIK (Naloxegol)
Opioid-induced constipation (OIC)
Approved
Approved September 16, 2014.
NLNK HyperAcute Pancreas - IMPRESS
Surgically resected pancreatic cancer
Phase 3
Phase 3 failed to meet primary endpoint - May 9, 2016.
NVAX RSV vaccine - Resolve trial
Respiratory Syncytial Virus (RSV) in older adults
Phase 3
Phase 3 released September 15, 2016 did not demonstrate efficacy.
NYMX NX-1207 Fexapotide
BPH
Phase 3
Phase 3 endpoints met in extension trial July 2015.
OCRX OCR-002 (IV)
Hepatic encephalopathy
Phase 2b
Phase 2b data released January 30, 2017 - primary endpoint not met.
OCUL DEXTENZA
Ocular inflammation and pain following cataract surgery
CRL
CRL July 25 2016 - due to manufacturing deficiencies.
OMED Demcizumab plus Abraxane - YOSEMITE
First-line pancreatic cancer
Phase 2
Phase 2 data released April 10, 2017 - primary endpoint not met.
OMED Demcizumab plus carboplatin and pemetrexed - DENALI
First-line non-squamous Non-small cell lung cancer (NSCLC)
Phase 2
Announced November 1, 2016 that it has discontinued further enrollment. Phase 2 data released May 8, 2017 - endpoints not met.
OMED Tarextumab - PINNACLE trial
Small cell lung cancer
Phase 2
Phase 2 data released April 17, 2017 - primary endpoint not met.
OMER Omidria
Cataract surgery
Approved
Approved June 2, 2014.
ONCE SPK-9001
Hemophilia B
Phase 1/2
Phase 1/2 updated data at ASH 2016. Further update released April 6, 2017.
OPHT Fovista
Wet age-related macular degeneration (Wet-AMD)
Phase 3
Phase 3 data released December 12, 2016 - primary endpoint not met.
OPK RAYALDEE
Secondary hyperparathyroidism (SHPT) in chronic kidney disease (CKD) and vitamin D insufficiency - Stage 3 and 4
CRL
CRL issued March 30, 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.
OREX Contrave
Obesity
CRL
CRL issued February 1, 2011.
OREX Contrave
Obesity
Approved
Approved September 11, 2014.
OTIC OTIPRIO (ciprofloxacin otic suspension)
Ear Tube Placement Surgery
Approved
Approved December 11, 2015.
PBMD IMP321
Breast cancer
Phase 2b
Phase 2b interim data released December 22, 2016.
PCRX EXPAREL
Single-dose injection femoral nerve block for total knee arthroplasty surgery
CRL
CRL March 2, 2015.
PGNX 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.
PGNX Oral RELISTOR
Chronic pain
Approved
Approved July 19 2016.
PGNX Subcutaneous RELISTOR
Chronic pain
PDUFA
Approved September 29, 2014.
PLX AIR DNase (PRX-110)
Cystic Fibrosis
Phase 2
Phase 2 interim data released January 3, 2017.
PLX AIR DNase (PRX-110)
Cystic Fibrosis
Phase 2
Phase 2 interim data released January 3, 2017. Full data released April 12, 2017. Company noted data were positive but results showed efficacy down on January data.
PRTK Omadacycline
Community-acquired bacterial pneumonia (CABP)
Phase 3
Phase 3 data released April 3, 2017 - all endpoints met.
PRTK Sarecycline
Severe acne vulgaris
Phase 3
Phase 3 data released March 27 2017 - primary endpoints met. NDA filing due 2H 2017.
PRTO Vonapanitase (PRT-201) PATENCY-1
Chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF)
Phase 3
Phase 3 data released December 13, 2016 - primary endpoint not met.
PSDV Iluvien
Diabetic macular edema
CRL
CRL November 11, 2011.
PSDV Iluvien
Diabetic macular edema
CRL
Second CRL October 18, 2013.
PSDV Iluvien
Diabetic macular edema
Approved
Approved Sept 26 2014.
PTCT Translarna
Nonsense mutation cystic fibrosis
Phase 3
Phase 3 data released March 2, 2017 - endpoints not met. Development to be discontinued.
PTLA Betrixaban (The APEX Study)
Venous thromboembolism (VTE) Prevention
Phase 3
Phase 3 data released March 24, 2016 did not meet primary endpoint.
PTLA Andexanet alfa
Factor Xa inhibitor reversal agent
CRL
CRL August 17 2016.
PTN Rekynda (Bremelanotide)
Female sexual dysfunction (FSD)
Phase 3
Phase 3 data released November 1, 2016.
PTX TREXIMET
Adolescent migraine patients
Approved
Approved May 15, 2015.
RARE rhGUS
Mucopolysaccharidosis 7 (MPS 7)
Phase 3
Phase 3 data released July 2016. Primary endpoint hit. Secondary missed.
RARE KRN23 Burosumab
X-Linked Hypophosphatemia (XLH) - adult
Phase 3
Phase 3 data released April 18, 2017 - primary endpoint met, two of three secondary endpoints met (missed on pain).
RDHL RHB-105
H. pylori
Phase 3
Phase 3 final data released March 8, 2016.
REPH Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery
Phase 3
Phase 3 data from second trial released November 28, 2016. Primary endpoint met.
RMTI Soluble Ferric Pyrophosphate
Iron deficiency
Approved
Approved January 26, 2015.
RTTR 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.
RVNC RT001 - REALISE 1
Lateral Canthal (Crow’s Feet) Lines
Phase 3
Phase 3 endpoints not met June 13, 2016.
RVNC RT002
Cervical dystonia
Phase 2
Phase 2 additional data released May 18, 2017 with oral presentation June 8, 2017 at International Congress of Parkinson's Disease and Movement Disorders.
SGEN ADCETRIS - AETHERA
Post-transplant Hodgkin lymphoma (HL) cancer
Approved
Approved August 17, 2015 under priority review.
SGYP Trulance (plecanatide)
Chronic idiopathic constipation (CIC)
Approved
Approved January 19, 2017.
SPPI EVOMELA (melphalan) for Injection
Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma
CRL
CRL issued October 23, 2015.
SPPI EVOMELA (melphalan) for Injection
Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma
Approved
Approved March 15 2016.
SPPI QAPZOLA - Apaziquone
Bladder cancer
Phase 3
Failed to meet primary endpoint in Phase 3 trial - Apr 2012. PDUFA date December 11 2016. Advisory Committee Meeting September 14 2016 voted 14 - 0 against approval. CRL issued November 17, 2016. Considering further trials.
SPPI Belinostat
Peripheral T-Cell Lymphoma cancer
PDUFA
Approved July 7, 2014.
SRPT Eteplirsen
Duchenne muscular dystrophy
Approved
Approved September 19, 2016.
SUPN Trokendi
Epilepsy
Approved
Approved Aug 19, 2013.
TCON TRC105 and Avastin
Glioblastoma - cancer
Phase 2
Phase 2 data released February 9, 2017 - primary endpoint not met.
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.
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.
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.
TSRO Niraparib - NOVA (Niraparib Ovarian)
Cancer - ovarian
Phase 3
Phase 3 data met endpoints - June 2016.
TSRO Niraparib - NOVA (Niraparib Ovarian)
Cancer - ovarian
Approved
PDUFA date under priority review was June 30, 2017. Approved March 27, 2017.
TSRO Rolapitant
Prevention of chemotherapy induced nausea and vomiting, or CINV in HEC patients
Approved
Approved September 2, 2015.
TTNP Probuphine
Opioid dependence
CRL
CRL issued April 30, 2013.
TTNP Probuphine
Opioid dependence
Approved
CRL issued April 30, 2013. Approved May 26, 2016.
VCEL MACI
Focal chondral cartilage defects in the knee.
Approved
FDA Approval announced December 13, 2016 ahead of PDUFA date of January 3 2017.
VNDA Fanapt
Supplemental New Drug Application (sNDA) as a maintenance treatment of schizophrenia in adults.
Approved
Approved May 26, 2016.
VNDA HETLIOZ (tasimelteon)
Insomnia
Approved
Approved January 31, 2014.
VRTX VX-150
Osteoarthritis
Phase 2
Phase 2 primary endpoint met - January 25, 2017.
VRTX KALYDECO (ivacaftor)
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
CRL
CRL issued February 5, 2016.
VRTX Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation
Approved
Approved July 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.
VTL VTI-308
Alcohol-induced liver decompensation, or AILD
Phase 3
Phase 3 VTI-208 trial did not meet endpoints - August 21, 2015.
VTVT TTP399 - AGATA
Type 2 Diabetes
Phase 2
Phase 2 data released August 10, 2016. Primary endpoint met.
VTVT TTP273 - LOGRA
Type 2 Diabetes
Phase 2
Phase 2 primary endpoint met - December 14, 2016.
VVUS Avanafil
Erectile dysfunction
Approved
Approved April 27, 2012.
XBIT Xilonix
Colorectal cancer
Phase 3
Phase 3 interim analysis announced February 21, 2017 - trial to continue as planned.
XENE XEN801
Acne
Phase 2
Phase 2 data released March 24 2017 - endpoints not met.
ZGNX Zohydro ER
Moderate to severe pain
Approved
Approved October 25, 2013.
AAAP Somakit-TATE
Neuroendocrine tumor diagnosis
Approved
Approved June 1, 2016.
ABUS ARB-1467
Hepatitis B (HBV)
Phase 2
Phase 2 single dose data released September 2016.
ABUS ARB-1467
Hepatitis B (HBV)
Phase 2
Phase 2 single dose data released September 2016, multiple dose data released December 12, 2016.
ACAD Pimavanserin
Parkinson’s disease psychosis (PDP)
Approved
Approved April 29, 2016.
ACOR Dalfampridine
Post stroke deficits
Phase 3
Phase 3 data released November 21, 2016 - failed to show efficacy, development to be discontinued.
ACOR CVT-301
OFF episodes of Parkinson’s disease (PD)
Phase 3
Phase 3 data released February 9, 2017 - primary endpoint met.
ACRS A-101
Seborrheic keratosis (SK)
Phase 3
Phase 3 trial met endpoints - November 15, 2016.
ACRX ARX-04 (SAP303)
Moderate-to-severe acute pain following a surgical procedure
Phase 3
Phase 3 data released August 15, 2016.
ACRX Zalviso
Post-operative pain following open abdominal surgery and hip or knee replacement surgery
CRL
CRL issued July 25 2014.
ADHD MDX (Metadoxine Extended Release (MG01CI))
Adult ADHD
Phase 3
Phase 3 data released January 17, 2017 - failed to meet primary endpoint.
ADMA RI-002
Primary Immune Deficiency Diseases
CRL
CRL issued July 29 2016. Third-party manufacturing issues cited.
ADMP Epinephrine Pre-filled Syringe (PFS)
Emergency treatment of anaphylaxis.
CRL
CRL issued March 27 2015.
ADMP Epinephrine Pre-filled Syringe (PFS)
Emergency treatment of anaphylaxis.
CRL
CRL issued March 27 2015. CRL issued again June 6, 2016.
ADMS Namzaric
Moderate to severe dementia of the Alzheimer's type.
Approved
Approved December, 24 2014.
AERI Roclatan - Mercury trials
Glaucoma
Phase 3
Phase 3 Mercury 1 data released September 14, 2016. Primary endpoint met.
AEZS Macimorelin Acetate - Macrilen
Adult Growth Deficiency
CRL
CRL issued November 6, 2014.
AEZS Macimorelin Acetate - Macrilen
Adult Growth Deficiency
Phase 3
Phase 3 data released January 4, 2017 - trial failed.
AEZS Zoptrex
Endometrial cancer
Phase 3
Phase 3 trial did not meet primary endpoint - May 1, 2017.
AKAO Plazomicin - EPIC trial
Complicated urinary tract infections (cUTI)
Phase 3
Phase 3 data released December 12, 2016 - primary endpoint met.
ALKS ALKS 6428
Opioid dependence
Phase 3
Phase 3 trial did not meet endpoint - February 2017 (exact date unsure).
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.
ALKS Aristada
Schizophrenia
Approved
Approved October 5, 2015.
ALNY Revusiran ENDEAVOUR
ATTR in patients with familial amyloidotic cardiomyopathy (FAC)
Phase 3
Phase 3 trial discontinued October 2016.
AMAG Feraheme
Adults with iron deficiency anemia (IDA)
CRL
CRL issued January 22, 2014.
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
ANTH Sollpura (liprotamase)
Cystic fibrosis who suffer from exocrine pancreatic insufficiency
Phase 3
Top-line data released December 27, 2016 - missed primary endpoint.
ARDM Pulmaquin - ORBIT-3 and ORBIT-4
Non-cystic fibrosis bronchiectasis (non-CF BE).
Phase 3
Phase 3 data released December 1, 2016. One of two trials met primary endpoint.
ARGS AGS-003 ADAPT Trial
Metastatic renal cell carcinoma (mRCC)
Phase 3
Phase 3 trial recommended trial be discontinued for futility - February 22, 2016.
ARLZ YOSPRALA (PA32540/PA8140)
Secondary prevention of cardiovascular disease in patients at risk for aspirin-induced gastric ulcers
CRL
CRL issued April 25, 2014.
ARLZ YOSPRALA (PA32540/PA8140)
Secondary prevention of cardiovascular disease in patients at risk for aspirin-induced gastric ulcers
CRL
CRL issued December 17, 2014.
ARLZ YOSPRALA (PA32540/PA8140)
Secondary prevention of cardiovascular disease in patients at risk for aspirin-induced gastric ulcers
Approved
Approval announced September 15, 2016.
ZFGN Beloranib
Prader-Willi Syndrome (PWS)
Phase 3
Phase 3 co-primary endpoints met.
ZGNX Abuse deterrent formulations of Zohydro ER
Moderate to severe pain
Approved
Approved January 30, 2015.
SPHS Topsalysin (PRX302)
Benign prostatic hyperplasia
Phase 3
Phase 3 primary endpoints met - November 10, 2015.
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.
TNXP TNX-102 SL
Fibromyalgia
Phase 3
Phase 3 data released September 6, 2016. Primary endpoint not met. Program discontinued
OPK Rolapitant
Prevention of chemotherapy induced nausea and vomiting, or CINV
Approved
Approved September 2, 2015.
OTIC OTIPRIO
Acute otitis externa
Phase 3
Phase 3 trial met primary endpoint - January 5, 2017.
RARE UX007 (triheptanoin)
Glucose Transporter Type-1 Deficiency Syndrome (Glut1 DS)
Phase 2
Phase 2 initiated March 2014. Data released March 22, 2017 - primary endpoint not met.
RIGL Fostamatinib
Immune Thrombocytopenic Purpura (ITP)
Phase 3
Phase 3 data from the first trial released August 30 2016. Primary endpoint met.
RIGL Fostamatinib
Immune Thrombocytopenic Purpura (ITP)
Phase 3
Phase 3 data from second trial did not meet the primary endpoint.
RPRX Enclomiphene
Secondary hypogonadism
CRL
CRL issued December 1, 2015.
LPTX CureXcell
Venous Leg Ulcers
Phase 3
Phase 3 endpoint not expected to be met - August 19, 2015.
GNMX NFC-1 - SAGA
mGluR mutation positive ADHD
Phase 2/3
Phase 2/3 data released March 20, 2017 - primary endpoint not met.
MRNS Ganaxolone
CDKL5 disorder
Phase 2
Phase 2 preliminary CDKL5 cohort data released January 23, 2017.
NBIX Elagolix
Endometriosis
Phase 3
First Phase 3 trial met both co-primary endpoints - January 8, 2015.
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.
NBIX INGREZZA
Tardive dyskinesia
Approved
Approved April 11, 2017.
NKTR Fovista
Wet-AMD
Phase 3
Phase 3 data released December 12, 2016 - primary endpoint not met.
GWPH Epidiolex
Dravet Syndrome
Phase 3
Top line Phase 3 data released March 14, 2016 met primary endpoint.
GWPH Epidiolex
Lennox-Gastaut syndrome (LGS)
Phase 3
First Phase 3 trial data released June 27, 2016.
GWPH Epidiolex
Lennox-Gastaut syndrome (LGS)
Phase 3
Second Phase 3 trial data met endpoint - September 2016.
GWPH Sativex
Spasticity due to cerebral palsy
Phase 2
Phase 2 trial did not meet primary endpoint - noted February 7, 2017.
DERM Glycopyrronium tosylate (DRM04)
Primary axillary hyperhidrosis
Phase 3
ATMOS-2 trial - co-primary endpoints and both secondary endpoints met. ATMOS-1 trial - one of the co-primary endpoints and both secondary endpoints met.
DRRX Remoxy
Chronic pain
CRL
CRL received June 24, 2011.
DRRX Remoxy
Chronic pain
CRL
CRL received September 26, 2016.
EARS AM-101 (Keyzilen) - TACTT2
Acute inner ear tinnitus
Phase 3
Phase 3 trial did not meet primary endpoints.
EGLT Egalet-001 (ARYMO ER)
Abuse-Deterrent Morphine
Approved
Approved January 9, 2017.
ENDP XIAFLEX
Edematous fibrosclerotic panniculopathy (“cellulite”)
Phase 2b
Phase 2b data released November 17, 2016. Met endpoints. Phase 3 planned.
FGEN Pamrevlumab (FG-3019)
Pancreatic cancer
Phase 2
Phase 2 abstract released January 17, 2017.
BLRX BL-8040
Mobilization and collection of bone marrow stem cells
Phase 2
Phase 2 trial initiated March 2016. Partial data released March 20, 2017.
BMRN Cerliponase alfa
Batten Disease
Approved
PDUFA date extended by three months to April 27 2017. Approval announced April 27, 2017.
BMRN Kyndrisa
Duchenne Muscular Dystrophy (DMD)
CRL
CRL issued January 14 2016.
BSTC XIAFLEX
Edematous fibrosclerotic panniculopathy (“cellulite”)
Phase 2b
Phase 2b data released November 17, 2016. Met endpoints
CARA IV CR845
Uremic pruritus
Phase 2/3
Phase 2/3 top-line data from Part A portion of trial released March 28, 2017 - endpoints met. Part B to be initiated later in 2017.
CERU CRLX101 and Avastin - RCC Trial
3rd and 4th line Relapsed Renal Cell Carcinoma cancer
Phase 2
Phase 2 data released August 2016 failed to meet primary endpoint
CLVS Rucaparib ARIEL2
Cancer - ovarian second line
Approved
Approved December 19, 2016. Original PDUFA date under priority review was February 23, 2017.
CRBP Anabasum (Resunab)
Systemic Sclerosis
Phase 2
Phase 2 data released November 14, 2016.
ADMS ADS-5102
Levodopa-Induced Dyskinesia
Phase 3
Phase 3 data released December 23, 2015.
ALIM Iluvien
Diabetic macular edema
CRL
CRL November 2011.
ALIM Iluvien
Diabetic macular edema
CRL
Second CRL October 17, 2013.
ALIM Iluvien
Diabetic macular edema
Approved
Approved September 26, 2014,
ANTH Blisibimod
Lupus
Phase 3
Phase 3 trial did not meet endpoints - November 10, 2016.
ARDX Tenapanor
Hyperphosphatemia in end-stage renal disease (ESRD) patients
Phase 3
Phase 3 released February 15, 2017 - primary endpoint met.
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.
BDSI BEMA Buprenorphine
Pain
Approved
Approved October 26, 2015.
ACAD Pimavanserin
Alzheimer’s disease psychosis
Phase 2
Phase 2 top-line data released December 20, 2016. Primary endpoint met. Key secondary endpoint missed.
ACRS A-101
Common warts (verruca vulgaris)
Phase 2
Phase 2 data released August 2016.