Breakthrough drugs for cystic fibrosis (CF)! Vertex's innovative triple therapy Trikafta treatment for children aged 6-11 years has been successful in Phase 3 research!

September 12, 2020 // -- Vertex Pharmaceuticals is a global leader in cystic fibrosis (CF) therapy.
, the company announced that a global Phase III study evaluating triple therapy Trikafta (elexacaftor/ivacaftor and ivacaftor) for children aged 6-11 years has been successfully completed.
the results, the company will submit a new drug supplement application (sNDA) to the FDA in the fourth quarter of 2020, followed by an application to other regulators around the world.
October 2019, Trikafta was approved by the FDA to treat at least one patient with an F508del mutation in the cystic fibrosis transfilm conduction regulatory factor (CFTR) gene, with a age of 12 years, in patients with 2 F508del mutations (F/F), or one patient with an F508del mutation and one minimum functional mutation (F/MF).
F508del mutation is the most common mutation that causes CF.
, the FDA has granted Trikafta priority qualification, fast-track eligibility, and breakthrough drug eligibility (BTD). Dr Carmen Bozic, Chief Medical Officer and Executive Vice President of Global Drug Development and Medical Affairs at
Vertex, said: "Our goal is to extend treatment eligibility to all patients who may benefit from this transformative drug, and the positive results of the Global Phase 3 study in children aged 6-11 enable us to move forward with this goal, and we look forward to the future Submit an sNDA within a few months and bring Trikafta to younger CF patients", a 24-week Open Label Global Phase 3 study of 66 CF children ages 6-11, assessed Trikafta's efficacy and safety.
the study, there were 2 F508del mutations in the CFTR gene, or one F508del mutation and one minimal functional mutation (F/MF).
end point of this study is safety and tolerance.
results showed that Trikafta was generally well-to-do at 24 weeks of treatment, and the safety data were consistent with those observed in previous Phase 3 studies.
In addition, clinically significant improvements were observed at several secondary efficacy endpoints over a 24-week period, including: percentage change in predicted one-second force exhalation (ppFEV1), sweat chloride, cystic fibrosis questionnaire-revision (CFQ-R) respiratory area score, body mass index (BMI) and other measurements.
the study showed that Trikafta's benefits-risk status in children aged 6-11 years were similar to those in Phase 3 studies that supported approval of the drug for PATIENTs 12 years of age and older.
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 transport of ions in 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, or about 50 percent of all CF patients, in the first three drugs.
and the newly approved triple therapy Trikafta can expand the treatment to 90% of CF patients worldwide.
June, Evaluate Phamra, a pharmaceutical market research organization, released a report predicting that Trikafta would become one of the world's best-selling TOP10 drugs by 2026, with sales of $8,739 million and a staggering compound annual growth rate of 54.3 percent for the period 2019-2026.
() Origin: Vertex Announces Positive Phase 3 Study for TRIKAFTA (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children 6-11 Years With Cystic Fibros to Support Products for Global Regulatory approval.