Breakthrough drugs for cystic fibrosis (CF)! Vertex's innovative triple therapy Kaftrio has been approved by the European Union to treat up to 90% of patients!

August 23, 2020 // -- Vertex Pharmaceuticals is a global leader in cystic fibrosis (CF) therapy.
announced recently that the European Commission (EC) has approved a 150mg joint drug treatment programme for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) for the treatment of 12 years of age In CF patients, specifically in patients with an F508del mutation and a minimal functional mutation (F/MF) in the cystic fibrosis transfilm conductivity regulator (CFTR) gene, or in patients with 2 F508del mutations (F/F).
this approval, for the first time in history, up to 10,000 cf patients in Europe who are 12 years of age, carrying an F508del mutation and a minimal functional mutation, are eligible for a In addition, CF patients aged 12 years and carrying two F508 mutations will also be eligible for a new triple treatment, which is currently eligible for another EU-approved CF drug from Vertex.
the U.S., the triple therapy was approved by the FDA in October 2019 under the brand name Trikafta (elexacaftor/tezacaftor/ivacaftor-ivacaftor) and is available for: C There is at least one patient with an F508del mutation in the F508del gene with an age of 12 years, specifically in patients with one F508del mutation and one with a minimal functional mutation, or with 2 patients with an F508del mutation.
, the FDA has granted Trikafta priority qualification, fast-track eligibility, and breakthrough drug eligibility (BTD).
F508del mutation is the most common mutation that causes CF.
in the U.S., Trikafta's approval is the first time that about 6,000 CF patients aged 12 with an F508del mutation and a minimal functional mutation (F/MF) have a treatment for the underlying cause of the disease.
addition, about 12,000 CF patients with one or two F508del mutations who are eligible for Vertex's three other FDA-approved CF medications are also eligible for Trikafta treatment.
the EUROPEAN Union approved Kaftrio based on the results of two Global Phase III studies: a 24-week placebo-controlled study in patients aged 12 years with an F508del mutation and a minimal functional mutation; The head study, conducted in CF patients aged 12 with two F508del mutations, compared triple therapy (ivacaftor/tezacaftor/elexacaftor) with tezacaftor/iafvactor.
two studies showed statistically and clinically significant improvements in the main endpoints (lung function, ppFEV1) and all key secondary endpoints.
of these two studies, ivacaftor/tezacaftor/elexacaftor and ivacaftor combined drug treatment programs were well-to-do.
Harry Heijerman, director of pulmonary medicine at the University medical centre in Udler, the Netherlands, said: "This triple therapy has been shown to have a significant impact on some outcome indicators in CF patients.
clinical data showed significant improvements in lung function and other important indicators in patients receiving triple therapy, such as chlorine content and quality of life of sweat measured by CFQ-R respiratory field scores.
now looking forward to seeing the effects of this drug in clinical practice.
Kewalramani, President and CEO of Vertex, said, "Today is a significant day for CF patients, their families and Vertex, and brings us one step closer to the ultimate goal of providing treatment to all CF patients.
I would like to thank our dedicated scientists, researchers and CF patients involved in our clinical trials for enabling this innovative drug to be approved in Europe.
this milestone would not have been possible without their dedication.
" cystic 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 currently has several CF drugs on the market: Kalydeco, Orkabi and Symkevi/Symdeko (tezacaftor/iafvactor) can treat about 40,000 patients worldwide, or about 50 percent of all CF patients.
new triple therapy Trikafta can expand the treatment to 90% of CF patients worldwide.
() Original source: European Commission approves KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in Union With Ivactor to Treat Cystic Fibrosis in People Ages 12 Years and Older.