The European Commission has approved expansion of the indication for Vertex Pharmaceuticals’ Kalydeco, to include children ages 2-5 with cystic fibrosis with one of 9 gating mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR), and to include people 18+ with CF with R117H mutation, the company said Wednesday.
The company notes that in Europe, approximately 125 children with CF ages 2 to 5 have one of the nine gating mutations included in Wednesday’s approval and approximately 350 adults with CF have an R117H mutation.
Cystic fibrosis is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe and Australia.
Vertex says it will now begin the country-by-country reimbursement approval processes for each new indication.
“These approvals bring us closer to our goal of developing new medicines to treat the underlying cause of cystic fibrosis for as many people as possible,” said Jeffrey Chodakewitz, M.D., Executive Vice President and Chief Medical Officer at Vertex.
Early treatment is critical for long-term outcomes
“We know that the progressive damage caused by cystic fibrosis can start at birth, so early treatment is critical to offering the best chance of improving long-term outcomes,” said Professor Jane Davies, M.D., of the Royal Brompton Hospital and Imperial College, London, and a lead Principal Investigator on the ivacaftor Phase 3 study in children ages 2 to 5.
“Today’s approval means that, for the first time, we’ll be able to treat the underlying cause of the disease in very young children, possibly even before they experience severe signs and symptoms of CF,” Davies said.
The European Commission’s Decision is based on previously announced results of a 24-week open-label Phase 3.
Expanding the use of Ivacaftor
The approval also includes an extension of weight-based dosing of ivacaftor to children ages 6 to 11 who weigh less than 25 kg, using the new oral granule formulation.
“Expanding the use of Ivacaftor will allow younger children with cystic fibrosis to benefit from earlier treatment of the underlying cause of their disease,” said Dr. Chodakewitz.
R117H mutation
“While people with an R117H mutation can exhibit a wide range of severity in their CF, once their disease begins to progress, lung function decline can be severe,” continued Dr. Chodakewitz.
“This approval is an important advance for adults with an R117H mutation who will now have a medicine to treat the underlying cause of their disease for the first time.”
The European Commission’s Decision is based on previously announced data from a Phase 3 study of ivacaftor that enrolled 69 people with CF who had an R117H mutation.