BOSTON–(BUSINESS WIRE)–December 21, 2020 — Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) expanded the eligibility for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include people with cystic fibrosis (CF) ages 12 years and older with certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that are responsive to Trikafta based on in vitro data. Symdeko (tezacaftor/ivacaftor and ivacaftor) and Kalydeco (ivacaftor) also received approvals to include additional responsive mutations in people with CF ages 6 years and older and age 4 months and older, respectively. These approvals allow more than 600 people with CF not previously eligible for these medicines an opportunity to potentially benefit from treatment that targets the underlying cause of their disease.
“The approval for expanded use of three of our CF medicines based on our well-established in vitro model is a testament to the relentless commitment of our scientists to reach our goal of developing treatments for all people with CF,” said Reshma Kewalramani, M.D., Chief Executive Officer and President, Vertex. “We remain as committed today to reaching every patient who might benefit from our medicines as when we first started out on this journey 20 years ago, and this important milestone now enables hundreds of people with CF access to a treatment option to address the underlying cause of their disease — many for the first time.”
Trikafta was previously approved for people with at least one F508del mutation and is now approved for 177 additional mutations; Symdeko is now approved for 127 additional mutations, for a total of 154 Symdeko-responsive mutations; and Kalydeco is now approved for an additional 59 mutations, for a total of 97 Kalydeco-responsive mutations. In addition, for certain people with CF who are currently eligible for Kalydeco, this approval allows them to also be eligible for Symdeko or Trikafta; and similarly, for those who are currently eligible for Symdeko, this approval allows them to also be eligible for Trikafta.
The full list of mutations for Trikafta, Symdeko and Kalydeco can be found within the updated full Prescribing Information for each respective product. In addition, people with CF and their families can search eligibility for Vertex CF medicines through vertextreatments.com.
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.
What is Kalydeco?
Kalydeco is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients age 4 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 4 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 or another mutation that is responsive to treatment with Trikafta. 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.
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. 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 11 consecutive years on Science magazine’s Top Employers list and a best place to work for LGBTQ equality by the Human Rights Campaign. 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. Reshma Kewalramani in this press release, statements regarding the eligible patient population for Trikafta, Symdeko and Kalydeco, our expectations regarding the number of patients newly eligible for Trikafta, Symdeko and Kalydeco, and statements regarding the potential benefits of Trikafta, Symdeko and Kalydeco. 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 data from the company’s development programs may not support registration or further development of its compounds due to safety, efficacy or other reasons, and other risks listed under the heading “Risk Factors” in Vertex’s most recent annual report and subsequent quarterly reports filed with the Securities and Exchange Commission (SEC) and available through the company’s website at www.vrtx.com and on the SEC’s website at www.sec.gov. 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.
Posted: December 2020
Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) FDA Approval History