VRTX Vertex Pharmaceuticals Incorporated
Upcoming Catalysts
| Drug | Stage | Catalyst Date |
|---|---|---|
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Elexacaftor, tezacaftor and ivacaftor
Children ages 6 to 11 years who have two F508del mutations and one F508del mutation and one minimal function mutation
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PDUFA
PDUFA
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TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
Children (6-11 years old) with cystic fibrosis (CF) with two copies of F508del mutation or one copy of F508del mutation and one minimal function mutation
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sNDA Filing
sNDA Filing
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CTX001
Beta-thalassemia
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Phase 1/2
Phase 1/2
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CTX001
Sickle cell disease
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Phase 1/2
Phase 1/2
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Drug Pipeline
| Drug | Stage | Notes |
|---|---|---|
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VX-814
alpha-1 antitrypsin (AAT) deficiency
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Phase 2
Phase 2
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Phase 2 trial has resumed at some sites following pause due to COVID-19 - July 30, 2020.
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VX-147
Focal segmental glomerulosclerosis (FSGS)
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Phase 2
Phase 2
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Phase 2 trial has been initiated - noted April 29, 2020.
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VX-864
Alpha-1 antitrypsin (AAT) deficiency
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Phase 1
Phase 1
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Phase 1 trial ongoing.
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VX-445 in combination with tezacaftor and ivacaftor
Cystic fibrosis (CF) who have one copy of the F508del mutation and one minimal function mutation and patients with two copies of the F508del mutation
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Approved
Approved
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FDA Approval announced October 21, 2019.
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Tezacaftor / ivacaftor
Cystic fibrosis - Two Copies of the F508del Mutation
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Approved
Approved
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Label expanded to now include all patients over 6 years old - June 21, 2019.
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Ivacaftor
Cystic Fibrosis (6-12 mths)
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Approved
Approved
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FDA Approval announced April 30, 2019.
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Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 1-2.
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Approved
Approved
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Approval announced August 15, 2018.
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Ivacaftor
Cystic fibrosis (CF) ages 2 to 5.
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Approved
Approved
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FDA Approval announced August 7, 2018.
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ORKAMBI
Cystic fibrosis (CF) ages 6-11 who have F508del mutation
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Approved
Approved
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Approved September 28, 2016.
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Tezacaftor (VX-661) / ivacaftor
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in a gating mutation
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Phase 3
Phase 3
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Phase 3 data released October 25, 2017 - primary endpoint not met.
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VX-661
Cystic fibrosis - one copy of the F508del mutation and a second mutation that results in minimal CFTR Function
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Phase 3
Phase 3
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Phase 3 trial terminated August 2016
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KALYDECO (ivacaftor)
Children ages 2 to 5 with cystic fibrosis who have the G551D or one of the eight additional gating mutations
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Approved
Approved
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Approved March 17, 2015.
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Lumacaftor and ivacaftor
Cystic fibrosis (CF) ages 12 and older who have two copies of the F508del mutation
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Approved
Approved
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Approved July 2, 2015.
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KALYDECO (ivacaftor)
Cystic fibrosis (CF) ages 2 and older who have one of 23 residual function mutations.
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CRL
CRL
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CRL issued February 5, 2016.
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KALYDECO
Cystic fibrosis (CF) ages 6 and older who have the R117H mutation
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Approved
Approved
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Approved December 29, 2014.
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Latest News
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Second collaboration between Moderna and Vertex based on Moderna's proprietary mRNA and LNP technology
Collaboration will leverage Vertex's investments and capabilities in genetic technologies for CF
Moderna to receive $75 million upfront, with potential for additional development, regulatory and commercial milestones and royalty payments
Moderna, Inc. (NASDAQ:MRNA) and Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) announced today a new strategic research collaboration and licensing agreement aimed at the discovery and development of lipid nanoparticles (LNPs) and mRNAs for the delivery of gene-editing therapies for the treatment of cystic fibrosis (CF). The three-year research collaboration initially will focus on the discovery and optimization of novel LNPs and mRNAs that can deliver gene-editing therapies to cells in the lungs, enabling functional cystic fibrosis transmembrane conductance regulator (CFTR) protein to be produced.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200916005915/en/
"We are pleased to enter into this second collaboration with Vertex aimed at delivering potentially novel treatments for patients with cystic fibrosis using gene editing," said Stéphane Bancel, Chief Executive Officer of Moderna. "Our first collaboration with Vertex to deliver mRNA coding for cystic fibrosis protein in lung cells is advancing well and this second collaboration aims at using Moderna's technologies to explore the use of gene editing in lung cells."
"Vertex's CFTR modulator therapies have the potential to treat the vast majority of CF patients and address the underlying cause of their disease. However, approximately 10 percent of patients do not produce any CFTR protein and so are unlikely to benefit from our existing medicines. Over the past 5 years, we have made important progress in our research efforts aimed at the creation of genetic therapies for CF, with the delivery of such therapies remaining the most significant technological and scientific challenge," said David Altshuler, M.D., Ph.D., Vertex's Executive Vice President, Global Research and Chief Scientific Officer. "The combination of Moderna's unique expertise in the discovery and manufacturing of novel LNP delivery systems and mRNA technologies, combined with Vertex's scientific, clinical and regulatory capabilities in CF, will accelerate the development of groundbreaking genetic therapies for people with CF and supports our commitment to developing therapies for all people living with CF."
Under the terms of the agreement, Moderna will conduct research activities to discover and optimize novel LNPs for the delivery of gene-editing therapies to lung cells for the treatment of CF. Moderna will receive $75 million upfront and will be eligible to receive up to $380 million in development, regulatory and commercial milestones, plus tiered royalties on any products that result from the collaboration. Moderna will be responsible for the discovery and manufacturing of LNPs and mRNA constructs encoding gene-editing endonucleases. Vertex will be responsible for providing other components of the gene-editing therapies to be formulated into LNPs, as well as subsequent preclinical and clinical development and potential commercialization efforts. This new collaboration adds to the existing relationship between Moderna and Vertex in their recently extended collaboration aimed at the discovery and development of mRNA therapeutics for the treatment of CF.
About Moderna
Moderna is advancing messenger RNA (mRNA) science to create a new class of transformative medicines for patients. mRNA medicines are designed to direct the body's cells to produce intracellular, membrane or secreted proteins that can have a therapeutic or preventive benefit and have the potential to address a broad spectrum of diseases. Moderna's platform builds on continuous advances in basic and applied mRNA science, delivery technology and manufacturing, providing the Company the capability to pursue in parallel a robust pipeline of new development candidates. Moderna is developing therapeutics and vaccines for infectious diseases, immuno-oncology, rare diseases, cardiovascular diseases, and autoimmune and inflammatory diseases, independently and with strategic collaborators.
Headquartered in Cambridge, Mass., Moderna currently has strategic alliances for development programs with AstraZeneca PLC and Merck & Co., Inc., as well as the Defense Advanced Research Projects Agency (DARPA), an agency of the U.S. Department of Defense; the Biomedical Advanced Research and Development Authority (BARDA), a division of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS) and the Coalition for Epidemic Preparedness Innovations (CEPI). Moderna has been named a top biopharmaceutical employer by Science for the past five years.
About Vertex Pharmaceuticals
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, Mr. Bancel's statements in the second paragraph of the press release, Dr. Altshuler's statements in the third paragraph of the press release, and the information provided regarding the future development of therapies to treat the underlying cause of CF, and the potential commercialization opportunities from those therapies. While Vertex and Moderna each believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the companies' beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that data may not support further development of the therapies subject to the collaboration due to safety, efficacy or other reasons, and other risks listed under the heading "Risk Factors" in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through Vertex's website at www.vrtx.com and on the SEC's website at www.sec.gov, as well as those risks listed under the heading "Risk Factors" in Moderna's most recent quarterly report on Form 10-Q filed with the SEC and in subsequent filings made by Moderna with the SEC, which are available on Moderna's website at www.modernatx.com and on the SEC's website at www.sec.gov. Except as required by law, Vertex and Moderna each disclaim any intention or responsibility for updating or revising any forward-looking statements contained in this press release in the event of new information, future developments or otherwise.
(VRTX – GEN)
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Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the European Medicines Agency (EMA) has validated a Type II Variation Marketing Authorization Application (MAA) for the expanded indication of KAFTRIO®* (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor to treat CF in patients ages 12 years and older with at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
If approved, eligible patients who have one copy of the F508del mutation and another CFTR mutation, such as a gating (F/G) or residual function (F/RF) mutation, will also be eligible for treatment. The MAA is supported by positive results from the global Phase 3 study (445-104) with KAFTRIO announced in July 2020. The application will now be reviewed by the Committee for Medicinal Products for Human Use (CHMP), which will issue an opinion to the European Commission regarding the potential approval for these patients.
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor is currently approved in Europe to treat people with CF ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the CFTR gene.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With ivacaftor
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg is approved in Europe for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KAFTRIO® is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. For complete product information, please see the Summary of Product Characteristics that can be found on https://www.ema.europa.eu/en/medicines/human/EPAR/kaftrio.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding the potential benefits of KAFTRIO, our expectations regarding the MAA, including, if approved, the patients that will become eligible for treatment, and our expectations regarding additional regulatory reviews, opinions and approvals and label expansions for our medicines. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities may not approve, or approve on a timely basis, the MAA, data from the company's development programs may not support registration, approval or further development of its compounds due to safety, efficacy or other reasons, risks related to approval and commercialization of our medicines, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
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Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the company has completed a global Phase 3 study of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in children ages 6 through 11 years old with cystic fibrosis (CF) who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation, and based on the results will submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2020, with additional global regulatory submissions to follow.
"Our aim is to extend eligibility to all patients who may benefit from this transformative medicine, and the positive results from the study in children ages 6 through 11 years old allows us to take another step forward toward this goal," said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex. "We are looking forward to filing an sNDA in the coming months and bringing TRIKAFTA to younger people with CF."
Bringing TRIKAFTA to Children Less than 12 Years of Age
The 24-week global Phase 3 open-label study evaluated the safety and efficacy of TRIKAFTA in 66 children ages 6 through 11 years old who have either two copies of the F508del mutation or one copy of the F508del mutation and one minimal function mutation. The primary endpoint of the study was safety and tolerability, and the results showed that TRIKAFTA was generally well tolerated and the safety data were consistent with those observed in previous Phase 3 studies. In addition, clinically meaningful improvements were seen across multiple secondary efficacy endpoints, including improvements in percent predicted forced expiratory volume in 1 second (ppFEV1), sweat chloride, Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory domain score, body mass index (BMI) and other measures through 24 weeks of treatment. The study showed the benefit-risk profile of TRIKAFTA in children with CF ages 6 through 11 years old was similar to that seen in people with CF ages 12 and older in the Phase 3 studies which have supported approval. Based on the results, Vertex will submit an sNDA to the U.S. FDA in the fourth quarter of 2020, with additional global regulatory submissions to follow.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
About TRIKAFTA®
TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients ages 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age. TRIKAFTA is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The approval of TRIKAFTA was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a 4-week Phase 3 study in 107 people with two F508del mutations (F/F).
U.S. INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) TABLETS
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.
Patients should not take TRIKAFTA if they take certain medicines, such as: antibiotics such as rifampin or rifabutin; seizure medicines such as phenobarbital, carbamazepine, or phenytoin; St. John's wort.
Before taking TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems, have or have had liver problems, are pregnant or plan to become pregnant because it is not known if TRIKAFTA will harm an unborn baby, or are breastfeeding or planning to breastfeed because it is not known if TRIKAFTA passes into breast milk.
TRIKAFTA may affect the way other medicines work, and other medicines may affect how TRIKAFTA works. Therefore, the dose of TRIKAFTA may need to be adjusted when taken with certain medicines. Patients should especially tell their doctor if they take: antifungal medicines including ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; antibiotics including telithromycin, clarithromycin, or erythromycin; other medicines including rifampin, rifabutin, phenobarbital, carbamazepine, phenytoin, and St. John's wort.
TRIKAFTA may cause dizziness in some people who take it. Patients should not drive a car, operate machinery, or do anything that requires alertness until they know how TRIKAFTA affects them.
Patients should avoid food or drink that contains grapefruit while they are taking TRIKAFTA.
TRIKAFTA can cause serious side effects, including:
High liver enzymes in the blood, which is a common side effect in people treated with TRIKAFTA. These can be serious and may be a sign of liver injury. The patient's doctor will do blood tests to check their liver before they start TRIKAFTA, every 3 months during the first year of taking TRIKAFTA, and every year while taking TRIKAFTA. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of the skin or the white part of the eyes; loss of appetite; nausea or vomiting; dark, amber-colored urine.
Abnormality of the eye lens (cataract) in some children and adolescents treated with TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with TRIKAFTA to look for cataracts.
The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.
These are not all the possible side effects of TRIKAFTA. Please click here to see the full Prescribing Information for TRIKAFTA.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Carmen Bozic in this press release, statements regarding the potential benefits of TRIKAFTA, our plans to make additional submissions to the FDA and other global regulatory agencies, including the timing of additional regulatory submissions, and our expectations regarding additional regulatory reviews and approvals of our medicines. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of risks and uncertainties that could cause actual events or results to differ materially from those expressed or implied by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities may not approve, or approve on a timely basis, the sNDA, data from the company's development programs may not support registration, approval or further development of its compounds due to safety, efficacy or other reasons, risks related to approval and commercialization of our medicines, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
(VRTX-GEN)
View source version on businesswire.com: https://www.businesswire.com/news/home/20200910005580/en/
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- More than 600 people with certain rare CF mutations could become newly eligible for TRIKAFTA, SYMDEKO or KALYDECO -
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor) and KALYDECO® (ivacaftor). These regulatory submissions are intended to expand the labels for TRIKAFTA, SYMDEKO and KALYDECO to include additional rare CFTR mutations, allowing people with cystic fibrosis (CF) not previously eligible for these medicines an opportunity to benefit from treatment that targets the underlying cause of their disease. In addition, these regulatory submissions may also allow certain people with CF who are currently eligible for KALYDECO to become eligible for SYMDEKO or TRIKAFTA and certain people currently eligible for SYMDEKO may become eligible for TRIKAFTA. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target action date of December 30, 2020. The regulatory submissions are based on data from an in vitro cell assay showing that these rare CFTR mutations respond to one or more of these CFTR modulator regimens.
"We have spent the last 20 years discovering, developing and bringing new medicines to thousands of people with CF, and the regulatory submissions announced today are an important next step in our commitment to bring transformative medicines to everyone living with this disease," said David Altshuler, M.D., Ph.D., Executive Vice President, Global Research and Chief Scientific Officer. "Using our well-established in vitro approach, we have been able to generate data providing evidence that people with certain rare mutations could benefit from treating the underlying cause of their disease with CFTR modulators."
These sNDAs are based on in vitro data from a validated cell assay model showing that many rare mutations in the CFTR gene are responsive to one or more of Vertex's medicines — KALYDECO, SYMDEKO and TRIKAFTA — beyond the mutations that are currently indicated for these therapies. Approximately 600 people in the U.S. who have certain rare CF mutations may benefit from TRIKAFTA, SYMDEKO or KALYDECO for the first time. In addition, more than 1,100 people with CF in the U.S. currently eligible for SYMDEKO or KALYDECO may have the option of an additional CFTR modulator. These regulatory submissions may allow certain people with CF who are currently eligible for KALYDECO to become eligible for SYMDEKO or TRIKAFTA and certain people currently eligible for SYMDEKO may become eligible for TRIKAFTA.
Data generated from this model, along with Phase 3 clinical data, have already led to the inclusion of nearly 30 additional ultra-rare and rare mutations in the U.S. for KALYDECO and SYMDEKO, including the first ever FDA approval based on in vitro data for a KALYDECO label expansion in patients with residual function CFTR mutations.
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR KALYDECO® (ivacaftor), SYMDEKO® (tezacaftor/ivacaftor and ivacaftor), and TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
What is KALYDECO?
KALYDECO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 months and older who have at least one mutation in their CF gene that is responsive to KALYDECO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if KALYDECO is safe and effective in children under 6 months of age.
What is SYMDEKO?
SYMDEKO is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 6 years and older who have two copies of the F508del mutation, or who have at least one mutation in the CF gene that is responsive to treatment with SYMDEKO. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if SYMDEKO is safe and effective in children under 6 years of age.
What is TRIKAFTA?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 12 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 12 years of age.
Patients should not take KALYDECO, SYMDEKO, or TRIKAFTA if they take certain medicines or herbal supplements, such as: the antibiotics rifampin or rifabutin; seizure medications such as phenobarbital, carbamazepine, or phenytoin; or St. John's wort.
Before taking KALYDECO, SYMDEKO, or TRIKAFTA, patients should tell their doctor about all of their medical conditions, including if they: have kidney problems; have or have had liver problems; are pregnant or plan to become pregnant because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA will harm an unborn baby; or are breastfeeding or planning to breastfeed because it is not known if KALYDECO, SYMDEKO, or TRIKAFTA passes into breast milk. Before taking KALYDECO, patients should tell their doctor if they drink grapefruit juice or eat grapefruit or Seville oranges.
KALYDECO, SYMDEKO, or TRIKAFTA may affect the way other medicines work, and other medicines may affect how KALYDECO, SYMDEKO, or TRIKAFTA work. Therefore, the dose of KALYDECO, SYMDEKO, or TRIKAFTA may need to be adjusted when taken with certain medications. Patients should especially tell their doctor if they take antifungal medications such as ketoconazole, itraconazole, posaconazole, voriconazole, or fluconazole; or antibiotics such as telithromycin, clarithromycin, or erythromycin.
KALYDECO, SYMDEKO, or TRIKAFTA can cause dizziness in some people who take it. Patients should not drive a car, use machinery, or do anything that needs them to be alert until they know how KALYDECO, SYMDEKO, or TRIKAFTA affects them.
Patients should avoid food or drink containing grapefruit or Seville oranges while taking KALYDECO. Patients should avoid food or drink containing grapefruit while taking SYMDEKO or TRIKAFTA.
KALYDECO, SYMDEKO, and TRIKAFTA can cause serious side effects, such as:
High liver enzymes in the blood have been reported in patients receiving KALYDECO, SYMDEKO, or TRIKAFTA. The patient's doctor will do blood tests to check their liver before starting treatment with KALYDECO, SYMDEKO, or TRIKAFTA, every 3 months during the first year of treatment, and every year while on treatment. For patients who have had high liver enzymes in the past, the doctor may do blood tests to check the liver more often. Patients should call their doctor right away if they have any of the following symptoms of liver problems: pain or discomfort in the upper right stomach (abdominal) area; yellowing of their skin or the white part of their eyes; loss of appetite; nausea or vomiting; or dark, amber-colored urine.
Abnormality of the eye lens (cataract) in some children and adolescents treated with KALYDECO, SYMDEKO, or TRIKAFTA. If the patient is a child or adolescent, their doctor should perform eye examinations before and during treatment with KALYDECO, SYMDEKO, or TRIKAFTA to look for cataracts.
The most common side effects of KALYDECO include headache; upper respiratory tract infection (common cold), which includes sore throat, nasal or sinus congestion, and runny nose; stomach (abdominal) pain; diarrhea; rash; nausea; and dizziness.
The most common side effects of SYMDEKO include headache, nausea, sinus congestion, and dizziness.
The most common side effects of TRIKAFTA include headache, diarrhea, upper respiratory tract infection (common cold) including stuffy and runny nose, stomach (abdominal) pain, inflamed sinuses, increase in liver enzymes, increase in a certain blood enzyme called creatine phosphokinase, rash, flu (influenza), and increase in blood bilirubin.
These are not all the possible side effects of KALYDECO, SYMDEKO, or TRIKAFTA. Please click the product link to see the full Prescribing Information for KALYDECO, SYMDEKO, or TRIKAFTA.
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes. For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com or follow us on Facebook, Twitter, LinkedIn, YouTube and Instagram.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Altshuler in this press release and statements regarding our expectations relating to the potential approval of TRIKAFTA, SYMDEKO, and KALYDECO for additional CFTR mutations, the FDA's target action date and information regarding the review process in the United States, the data supporting product approval, and the number of additional patients that may benefit from our medicines. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that regulatory authorities may not approve, or approve on a timely basis, the three sNDAs, that data from the company's submissions may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under Risk Factors in Vertex's annual report and quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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– For the first time, up to 10,000 people in Europe ages 12 years and older with one F508del mutation and one minimal function mutation will be eligible for a medicine that treats the underlying cause of cystic fibrosis –
– People 12 years of age and older who have two F508del mutations will also be eligible for the new triple combination regimen –
Vertex Pharmaceuticals Incorporated (NASDAQ:VRTX) today announced that the European Commission (EC) has granted marketing authorization for KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor to treat people with cystic fibrosis (CF) ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF), or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.
For the first time, up to 10,000 people in Europe ages 12 years and older with CF who have one F508del mutation and one minimal function mutation will be eligible for a CFTR modulator that treats the underlying cause of the disease. Approval of the triple combination regimen also expands the number of treatment options available to people ages 12 years and older with CF who have two copies of the F508del mutation, the most common CF-causing mutation worldwide.
"Today is a significant day for those with CF, their families and Vertex, and one that brings us one step closer towards our ultimate goal of discovering and developing treatments for all patients with CF," said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. "I would like to thank our dedicated scientists, as well as study investigators and people with CF who participated in our clinical trials to enable this innovative medicine to be approved in Europe today. Without their commitment, this milestone would not have been possible."
As a result of long-term reimbursement agreements in England, Denmark and the Republic of Ireland, and provisions for access in health care systems such as Germany, eligible patients in these countries will have access to the triple combination regimen in the upcoming weeks. Vertex is committed to working closely with national health authorities and governments in all other countries in Europe to secure access for eligible patients as quickly as possible.
Marketing authorization was based on the results of two global Phase 3 studies, which showed statistically significant and clinically meaningful improvements in lung function (primary endpoint) and all key secondary endpoints, in people with CF ages 12 years and older with one F508del mutation and one minimal function mutation or two F508del mutations in the CFTR gene. The triple combination regimen was generally well tolerated in both studies.
"The triple combination regimen has been shown to have a major impact on several outcome measures in people with CF," said Professor Harry Heijerman, Professor and Head of the Department of Pulmonology at University Medical Center Utrecht, Netherlands. "The clinical data showed significant improvements in lung function and other important measures, such as sweat chloride levels and quality of life as measured by the CFQ-R respiratory domain score, in patients treated with the triple combination therapy. I now look forward to seeing the impact of the medicine in clinical practice."
About Cystic Fibrosis
Cystic Fibrosis (CF) is a rare, life-shortening genetic disease affecting approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, GI tract, sinuses, sweat glands, pancreas and reproductive tract. CF is caused by a defective and/or missing CFTR protein resulting from certain mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to have CF. While there are many different types of CFTR mutations that can cause the disease, the vast majority of all people with CF have at least one F508del mutation. These mutations, which can be determined by a genetic test, or genotyping test, lead to CF by creating non-working and/or too few CFTR proteins at the cell surface. The defective function and/or absence of CFTR protein results in poor flow of salt and water into and out of the cells in a number of organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage in many patients that eventually leads to death. The median age of death is in the early 30s.
About KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a Combination Regimen With ivacaftor
KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in a combination regimen with ivacaftor 150 mg was developed for the treatment of cystic fibrosis (CF) in patients ages 12 years and older with one F508del mutation and one minimal function mutation (F/MF) or two F508del mutations (F/F) in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. KAFTRIO® is designed to increase the quantity and function of the F508del-CFTR protein at the cell surface. The EU submission for KAFTRIO® was supported by positive results of two global Phase 3 studies in people ages 12 years and older with CF: a 24-week Phase 3 study in 403 people with one F508del mutation and one minimal function mutation (F/MF) and a four-week Phase 3 study in 107 people with two F508del mutations (F/F).
About Vertex
Vertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has multiple approved medicines that treat the underlying cause of cystic fibrosis (CF) — a rare, life-threatening genetic disease — and has several ongoing clinical and research programs in CF. Beyond CF, Vertex has a robust pipeline of investigational small molecule medicines in other serious diseases where it has deep insight into causal human biology, including pain, alpha-1 antitrypsin deficiency and APOL1-mediated kidney diseases. In addition, Vertex has a rapidly expanding pipeline of genetic and cell therapies for diseases such as sickle cell disease, beta thalassemia, Duchenne muscular dystrophy and type 1 diabetes mellitus.
Founded in 1989 in Cambridge, Mass., Vertex's global headquarters is now located in Boston's Innovation District and its international headquarters is in London, UK. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia and Latin America. Vertex is consistently recognized as one of the industry's top places to work, including 10 consecutive years on Science magazine's Top Employers list and top five on the 2019 Best Employers for Diversity list by Forbes.
Special Note Regarding Forward-looking Statements
This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Dr. Reshma Kewalramani and Professor Harry Heijerman in this press release, statements regarding the eligible patient population in Europe, our expectations regarding the timing of access to the triple combination regimen across countries in Europe, and our plans to secure access to our medicine for additional patients in Europe. While Vertex believes the forward-looking statements contained in this press release are accurate, these forward-looking statements represent the company's beliefs only as of the date of this press release and there are a number of factors that could cause actual events or results to differ materially from those indicated by such forward-looking statements. Those risks and uncertainties include, among other things, that data from the company's development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, risks related to commercializing medicines in Europe, and other risks listed under Risk Factors in Vertex's annual report and subsequent quarterly reports filed with the Securities and Exchange Commission and available through the company's website at www.vrtx.com. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.
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