Vertex Pharmaceuticals is a global leader in cystic fibrosis (CF) therapy.
recently, the company announced that the U.S. Food and Drug Administration (FDA) has accepted its application for new complementary drugs (sNDA) to expand the triple therapy Trikafta (elexacaftor/tezacaftor/ivacaftor and the scope of use of cavtor) to include CFTR gene carrying at least one F508del mutation, or CFTR gene carrying a specific mutation and based on in vitro data to respond to Trikafta treatment of 6-11 year old CF patients.
FDA has granted the sNDA priority review and has designated a target date for the Prescription Drug User Charge Act (PDUFA) as June 8, 2021.
the U.S., Trikafta has been approved for use in patients ≥12-year-old CF who carry at least one F508del mutation, or the CFTR gene that has a specific mutation and responds to Trikafta therapy based on in vitro data.
The approval of the expansion, we will have the opportunity to treat the underlying causes of the disease with Trikafta early in life and potentially benefit approximately 1,500 CF children," said Carmen Bozic, executive vice president and chief medical officer of
first regulatory approval from Trikafta in 2019, we have been working tirelessly to bring the drug to patients waiting as soon as possible.
look forward to working with the agency in the coming months to review the application.
" 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.
also plans to file applications in the coming months for this age group in other markets such as Canada and Australia.
trikafta to expand application applications, based on data support from a global Phase 3 study.
the study was conducted in patients aged 6-11 with 2 copies of the F508del mutation, or 1 copy of the F508del mutation and one minimum functional mutation, to assess the efficacy and safety of Trikafta.
fibrosis (CF) is a rare, life-shortening genetic disease that affects about 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 certain mutations in the CFTR gene that cause defects or deficiencies in the function of the CFTR protein.
children must inherit two defective CFTR genes (one for each parent) to develop CF.
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.
CFTR protein usually regulates the ion transport of cell membranes, and genetic mutations can lead to the destruction or loss of protein product function.
when cell membrane ion transport is interrupted, the viscosity of the mucus coating of some organs thickens.
a major feature of the disease is the build-up of thick mucus in the respiratory tract, which leads to breathing difficulties, chronic recurrent infections of the lungs and recurrent lung damage, which eventually leads to death.
the median age of death for CF patients was in their mid-30s.
, Vertex has listed four CF drugs: Kalydeco( ivacaftor), Orkabi (lumacaftor/ivacaftor), Symdeko (tezacaftor/ivacaftor and The first three drugs treat about 40,000 patients worldwide, about 50 percent of all CF patients, in the first three drugs.
and Trikafta, the newly approved triple therapy, could expand to 90 percent of CF patients worldwide by the end of 2019.