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New medicine for cystic fibrosis (CF)! The US FDA approves the Vertex triple therapy Trikafta to expand the applicable population: treat children aged 6-11!
FDA approves Vertex Trikafta to expand treatment age to 6-11 chirldren! Vertex Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has approved the expansion of the use of triple therapy Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include at least one F508del mutation in the CFTR gene, or a specific mutation in the CFTR gene And based on in vitro data, 6-11-year-old CF patients who responded to Trikafta treatment.
The additional dosage strengths of Trikafta tablets (elexacaftor 50mg/tezacaftor 25mg/ivacaftor 37.5mg and ivacaftor 75mg) related to this approval are now available for patients.
In the United States, Trikafta was previously 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.
Trikafta’s expanded indication approval is based on data from a 24-week Phase 3 open-label multicenter study. The study included 66 CF patients aged 6-11 years old who carried 2 copies of the F508del mutation, or 1 copy of the F508del mutation and a minimal functional mutation. The study evaluated the safety, pharmacokinetics and efficacy of Trikafta.
The results show that Trikafta is generally well tolerated in this patient population, and the safety data are consistent with previous studies on patients 12 years of age and older. The entire data of the study was recently published in the international medical journal “American Journal of Respiratory and Critical Care Medicine.”
Vertex has also submitted Trikafta to the European Medicines Agency (EMA) and the British Medicines and Healthcare Products Agency (MHRA) for the treatment of children with CF between 6-11 years old. The company also plans to submit application documents for this age group in Switzerland, Australia, Israel and other markets in the next few months.
Carmen Bozic, MD, Executive Vice President and Chief Medical Officer of Vertex said: “Today’s approval is a key milestone in our efforts to provide drugs to treat the underlying cause of this devastating disease as early as possible. In the United States, with this expanded approval, there will be Approximately 1,500 children with CF are eligible for Trikafta treatment. We will continue to strive for approval of this expanded indication in other countries.”
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, the vast majority of 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, leading to difficulty breathing, 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.
(source:internet, reference only)