BOSTON - Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) accepted three supplemental New Drug Applications (sNDAs) for TRIKAFTA, SYMDEKO 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.
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. 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.
Contact:
Tel: 617-961-7163
Email: mediainfo@vrtx.com
VERTEX PHARMACEUTICALS INCORPORATED 
