\n– ALYFTREK<\/i>\u2122<\/sup> is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies –<\/i><\/p>\n \n– In head-to-head clinical trials, ALYFTREK was non-inferior on ppFEV1<\/sub> and further decreased sweat chloride compared to TRIKAFTA\u00ae <\/sup>–<\/i><\/p>\n BOSTON–(BUSINESS WIRE<\/a>)–Vertex Pharmaceuticals Incorporated<\/a> (Nasdaq: VRTX) today announced that the U.S. Food and Drug Administration (FDA) has approved ALYFTREK (vanzacaftor\/tezacaftor\/deutivacaftor), a once-daily next-in-class triple combination cystic fibrosis transmembrane conductance regulator (CFTR) modulator for the treatment of cystic fibrosis (CF) in people 6 years and older who have at least one F508del<\/i> mutation or another mutation in the CFTR<\/i> gene that is responsive to ALYFTREK. See below for Important Safety Information, including a Boxed Warning.<\/p>\n \n\u201cALYFTREK is our fifth CFTR modulator to secure FDA approval and represents another significant milestone in our journey to serially innovate and to improve the lives of people living with cystic fibrosis,\u201d said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. \u201cOur north star for more than 20 years has been to address the underlying cause of cystic fibrosis, treat more people with this disease, and bring more people to normal levels of CFTR function \u2014 ALYFTREK, with once-daily dosing, efficacy in 31 additional mutations, and lower sweat chloride levels than TRIKAFTA, is another step in achieving this goal.\u201d<\/p>\n \nThis approval is based on the most comprehensive Phase 3 pivotal program ever conducted in CF, including more than 1,000 patients across more than 20 countries and more than 200 sites. These data were previously released at the conclusion of the studies<\/a> and presented at the North American Cystic Fibrosis Conference<\/a> in September of this year. The Phase 3 studies in people with CF ages 12 years and older met their primary endpoint (non-inferiority on absolute change from baseline in ppFEV1<\/sub> compared to TRIKAFTA) and all key secondary endpoints (including absolute change from baseline in sweat chloride [SwCl] compared to TRIKAFTA). In the Phase 3 study of children with CF ages 6-11 years, ALYFTREK demonstrated safety, the primary endpoint. Secondary endpoints, such as absolute change from baseline in ppFEV1<\/sub> and absolute change from baseline in SwCl, were presented, supporting the benefit of ALYFTREK in this age group. ALYFTREK was generally well tolerated across all studies.<\/p>\n \n\u201cIn Phase 3 clinical trials, across a broad range of genotypes, once-daily ALYFTREK demonstrated non-inferiority to TRIKAFTA in ppFEV1<\/sub> response and statistically significant improvement in SwCl, a welcomed advancement for the treatment of CF,\u201d said Claire L. Keating, M.D., Co-Director of the Gunnar Esiason Adult Cystic Fibrosis and Lung Program at Columbia University and investigator in the ALYFTREK clinical trial program. \u201cALYFTREK has the potential to improve the care of patients with CF.\u201d<\/p>\n \nALYFTREK is the first, once-daily CFTR modulator. In a recent survey, approximately 75% of physicians reported that more convenient dosing is a very high unmet need for people with CF. Specifically, people with CF will have the added benefit from a once-daily dosing regimen, given the need to take CFTR modulators with fat-containing food. ALYFTREK also offers a potentially transformative option for approximately 150 people with CF in the U.S. with one of 31 mutations who are now eligible for a CFTR modulator for the first time.<\/p>\n \nALYFTREK was also submitted to global health authorities and is under regulatory review in the European Union, the United Kingdom, Canada, Switzerland, Australia and New Zealand.<\/p>\n About Cystic Fibrosis<\/b><\/p>\n \nCystic fibrosis (CF) is a rare, life-shortening genetic disease affecting more than 92,000 people globally. CF is a progressive, multi-organ disease that affects the lungs, liver, pancreas, GI tract, sinuses, sweat glands and reproductive tract. CF is caused by a defective and\/or missing CFTR protein resulting from certain mutations in the CFTR<\/i> gene. Children must inherit two defective CFTR<\/i> genes \u2014 one from each parent \u2014 to have CF, and these mutations can be identified by a genetic test. While there are many different types of CFTR<\/i> mutations that can cause the disease, the vast majority of people with CF have at least one F508del<\/i> mutation. CFTR <\/i>mutations lead to CF by causing CFTR protein to be defective or by leading to a shortage or absence of CFTR protein 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, chronic lung infections and progressive lung damage that eventually leads to death for many patients. The median age of death is in the 30s, but with treatment, projected survival is improving.<\/p>\n Learn more<\/a> about the importance of sweat chloride (SwCl) in cystic fibrosis.<\/p>\n \nToday Vertex CF medicines are treating over 68,000 people with CF across more than 60 countries on six continents. This represents 2\/3 of the diagnosed people with CF eligible for CFTR modulator therapy.<\/p>\n ALYFTREK U.S. INDICATIONS<\/b><\/p>\n \nALYFTREK is indicated for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.<\/p>\n \nIf the patient\u2019s genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one indicated mutation.<\/p>\n IMPORTANT SAFETY INFORMATION<\/b><\/p>\n BOXED WARNING: DRUG-INDUCED LIVER INJURY AND LIVER FAILURE<\/b><\/p>\n Elevated transaminases have been observed in patients treated with ALYFTREK. Cases of serious and potentially fatal drug-induced liver injury and liver failure were reported in patients taking a fixed-dose combination drug containing elexacaftor, tezacaftor, and ivacaftor, which contains the same or similar active ingredients as ALYFTREK. Liver injury has been reported within the first month of therapy and up to 15 months following initiation of elexacaftor\/tezacaftor\/ivacaftor.<\/b><\/p>\n Assess liver function tests (ALT, AST, alkaline phosphatase, and bilirubin) in all patients prior to initiating ALYFTREK, every month during the first 6 months of treatment, every 3 months for the next 12 months, and at least annually thereafter. Consider more frequent monitoring for patients with a history of liver disease or elevated liver function tests (LFTs) at baseline.<\/b><\/p>\n Interrupt ALYFTREK for significant elevations in LFTs or in the event of signs or symptoms of liver injury. Consider referral to a hepatologist. Follow patients closely with clinical and laboratory monitoring until abnormalities resolve. If resolved, resume treatment only if benefit is expected to outweigh risk. Closer monitoring is advised after resuming ALYFTREK.<\/b><\/p>\n ALYFTREK should not be used in patients with severe hepatic impairment (Child-Pugh Class C). ALYFTREK is not recommended in patients with moderate hepatic impairment (Child-Pugh Class B) and should only be considered when there is a clear medical need, and benefit outweighs risk. If used, monitor patients closely.<\/b><\/p>\n WARNINGS AND PRECAUTIONS<\/b><\/p>\n Drug-Induced Liver Injury and Liver Failure<\/b><\/p>\n Hypersensitivity Reactions, Including Anaphylaxis<\/b><\/p>\n Patients Who Discontinued or Interrupted Elexacaftor-, Tezacaftor-, or Ivacaftor-Containing Drugs Due to Adverse Reactions<\/b><\/p>\n Reduced Effectiveness with Concomitant Use With CYP3A Inducers<\/b><\/p>\n Adverse Reactions with Concomitant Use With CYP3A Inhibitors<\/b><\/p>\n Cataracts<\/b><\/p>\n ADVERSE REACTIONS<\/b><\/p>\n Serious Adverse Reactions<\/b><\/p>\n Most Common Adverse Reactions<\/b><\/p>\n USE IN SPECIFIC POPULATIONS<\/b><\/p>\n Pediatric Use<\/b><\/p>\n Please <\/b>click here<\/a> to see the full U.S. Prescribing Information, including Boxed WARNING for ALYFTREK.<\/b><\/p>\n About Vertex<\/strong><\/p>\n \nVertex is a global biotechnology company that invests in scientific innovation to create transformative medicines for people with serious diseases. The company has approved medicines that treat the underlying causes of multiple chronic, life-shortening genetic diseases \u2014 cystic fibrosis, sickle cell disease and transfusion-dependent beta thalassemia \u2014 and continues to advance clinical and research programs in these diseases. Vertex also has a robust clinical pipeline of investigational therapies across a range of modalities in other serious diseases where it has deep insight into causal human biology, including acute and neuropathic pain, APOL1-mediated kidney disease, IgA nephropathy, primary membranous nephropathy, autosomal dominant polycystic kidney disease, type 1 diabetes and myotonic dystrophy type 1.<\/p>\n \nVertex was founded in 1989 and has its global headquarters in Boston, with international headquarters in London. Additionally, the company has research and development sites and commercial offices in North America, Europe, Australia, Latin America and the Middle East. Vertex is consistently recognized as one of the industry’s top places to work, including 15 consecutive years on Science magazine’s Top Employers list and one of Fortune\u2019s 100 Best Companies to Work For. For company updates and to learn more about Vertex’s history of innovation, visit www.vrtx.com<\/a> or follow us on LinkedIn<\/a>, Facebook<\/a>, Instagram<\/a>, YouTube<\/a> and X<\/a>.<\/p>\n Special Note Regarding Forward-Looking Statements<\/b><\/p>\n \nThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements made by Reshma Kewalramani, M.D., and Claire L. Keating, M.D., in this press release, statements regarding the eligible patient population for ALYFTREK, expectations for ALYFTREK regulatory submissions to global health authorities, and statements regarding the potential benefits of ALYFTREK, including for patients eligible for a CFTR modulator for the first time. 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 risks listed under the heading \u201cRisk Factors\u201d in Vertex’s annual report and in subsequent filings filed with the Securities and Exchange Commission and available through the company’s website at www.vrtx.com<\/a> and www.sec.gov<\/a>. You should not place undue reliance on these statements. Vertex disclaims any obligation to update the information contained in this press release as new information becomes available.<\/p>\n \n(VRTX-GEN)<\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20241220133127\/en\/<\/a><\/span><\/p>\n Vertex Pharmaceuticals Incorporated\n
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Investors:
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Susie Lisa, CFA: +1 617-341-6108
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Manisha Pai: +1 617-961-1899<\/p>\n
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