Vertex Pharmaceuticals is a global leader in the treatment of cystic fibrosis (CF). Recently, the company announced that the U.S. Food and Drug Administration (FDA) has accepted its supplementary new drug application (sNDA) to expand the use of triple therapy Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include at least one F508del in the CFTR gene. 6-11-year-old CF patients who have a mutation or a specific mutation in the CFTR gene and respond to Trikafta treatment based on in vitro data. The FDA has granted the sNDA a priority review and has designated the Prescription Drug User Fee Act (PDUFA) target date as June 8, 2021.
In the United States, Trikafta has been approved for use in CF patients ≥ 12 years of age who carry at least one F508del mutation or a specific mutation in the CFTR gene and based on in vitro data that the mutation responds to Trikafta treatment.
Carmen Bozic, MD, Executive Vice President and Chief Medical Officer of Vertex said: "If expanded use is approved, we will have the opportunity to use Trikafta to treat the underlying cause of the disease early in life, and may benefit approximately 1,500 children with CF. First received since 2019 Since Trikafta's regulatory approval, we have been working tirelessly to bring this medicine to those waiting patients as soon as possible. We look forward to working with the agency to review the application in the coming months."
Vertex also plans to submit a marketing authorization application (MAA) change to the European Medicines Agency (EMA) in the first half of 2021 for CF children aged 6-11. The company also plans to submit application documents for this age group in other markets such as Canada and Australia in the coming months. Trikafta's application for expanded application is supported by data from a global Phase 3 study. The study was carried out in 6-11-year-old CF patients with 2 copies of F508del mutation, or 1 copy of F508del mutation and one minimal functional mutation, and evaluated the efficacy and safety of Trikafta.
Cystic fibrosis (CF) is a rare, life-shortening genetic disease that affects approximately 75,000 people worldwide. CF is a progressive, multi-system disease that affects the lungs, liver, gastrointestinal tract, sinuses, sweat glands, pancreas, and reproductive tract. CF is caused by some mutations in the CFTR gene that cause CFTR protein function defects or deletions. Children must inherit 2 defective CFTR genes (one for each parent) to develop CF.
Although there are many different types of CFTR mutations that can cause disease, most CF patients have at least one F508del mutation. These mutations can be determined by genetic testing or genotyping. The CFTR protein usually regulates ion transport in the cell membrane, and gene mutations can cause the destruction or loss of the function of the protein product. When ion transport in the cell membrane is interrupted, the viscosity of the mucus coating on certain organs will thicken. One of the main features of the disease is the accumulation of thick mucus in the respiratory tract, which leads to breathing difficulties, chronic recurrent lung infections, and progressive lung damage, eventually leading to death. The median age of death for CF patients is in their early 30s.
Up to now, Vertex has listed 4 CF drugs: Kalydeco (ivacaftor), Orkabi (lumacaftor/ivacaftor), Symdeko (tezacaftor/ivacaftor and ivacaftor), Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor), the first 3 drugs can treat There are about 40,000 patients worldwide, accounting for about 50% of all CF patients. The newly approved triple therapy Trikafta at the end of 2019 can expand the treatment range to 90% of CF patients worldwide.
The pharmaceutical market research organization EvaluatePhamra released a report predicting that Trikafta will become one of the world's best-selling TOP10 drugs in 2026, with sales reaching 8.739 billion U.S. dollars, and a compound annual growth rate of 54.3% during 2019-2026.