Kalydeco approved in EU for CF children as young as 1 month
Treatment also expanded in United Kingdom, United States for this age
The European Commission (EC) has expanded the approval of Kalydeco (ivacaftor) for the treatment of infants with cystic fibrosis (CF) as young as 1 month with at least one CFTR gating mutation.
The approval makes Kalydeco, marketed by Vertex Pharmaceuticals, the first and only CFTR modulator approved in the European Union (EU) for this patient population. The therapy is also available to month-old infants in the U.S.
“Treating CF early in life can potentially slow the progression of the disease, which is why it is so important to start treatment from a very young age,” Carmen Bozic, MD, executive vice president of global medicines development and medical affairs and chief medical officer at Vertex, said in a company press release.
CFTR is a protein that acts like a gate to control the flow of chloride ions into and out of cells, helping maintain the balance of water and salt on tissue surfaces.
People with CF carry inherited mutations in the CFTR gene, which lead to a missing or defective CFTR protein. Gating mutations are those that impair the CFTR protein’s gatelike function, blocking the flow of chloride and causing an abnormal buildup of thick mucus in various organs.
CFTR modulators are a class of medicines designed to improve the production or function of faulty CFTR protein. Kalydeco, known as a CFTR potentiator, enhances CFTR protein activity by keeping the gate open longer at the cell surface, increasing the flow of water and salt and reducing the thickness of mucus.
The approval update
Kalydeco initially gained approval in Europe for patients aged 2 and older, with an extension in 2018 to include 1-year-olds. Eventually, the EC further expanded its authorization to encompass infants as young as 6 months and, later, to those as young as 4 months with CF caused by specific mutations.
This label extension applies to European children as young as a month with one of the following 10 CFTR gating mutations: R117H, S1251N, S1255P, S549N, S549R, G551D, G1244E, G1349D, G178R, or G551S.
EC’s decision comes on the heels of an endorsement by the Committee for Medicinal Products for Human Use, an arm of the European Medicines Agency.
Under existing access agreements, eligible patients in Austria, the Czech Republic, Denmark, Ireland, the Netherlands, Norway, and Sweden will be able to receive Kalydeco soon after the EC’s regulatory expansion. Vertex says it will work with reimbursement authorities across the EU to ensure all other eligible patients receive the treatment, too. In the U.K., eligible infants as young as a month also will have access to this expanded indication.
Data from the Phase 3 ARRIVAL (NCT02725567) clinical trial supported the label extension. According to Vertex, Kalydeco’s safety profile among CF patients younger than 2 years was similar to that reported in older children and adults.
“Today’s approval is an important milestone for the cystic fibrosis community,” Bozic said.
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