US approval for Orkambi in children with CF aged 2-5

First US approval for Sanofi’s rare blood disorders franchise
Credit: Castleski

Vertex Pharmaceuticals has secured approval from US regulators for Orkambi as the first medicine to treat the underlying cause of cystic fibrosis (CF) for changed aged 2-5.

“For the first time, children ages 2 through 5 who have the most common form of CF have a treatment for the underlying cause of their disease,” said Reshma Kewalramani, Executive Vice President and Chief Medical Officer at Vertex.

“We believe it is important to treat the underlying cause of the disease as early as possible and this approval is another significant milestone in our journey to bring effective medicines to all people living with CF.”

This FDA approval is based on a Phase 3 open-label safety study in 60 patients that showed treatment with Orkambi was generally safe and well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6 years and older. Improvements in sweat chloride, a secondary endpoint, were observed at week 24.

Researchers also saw changes in key growth parameters, which were also secondary endpoints in the study.

Orkambi was already approved in the US for the treatment of CF in patients ages 6 and older who have two copies of the F508del-CFTR mutation.

A Marketing Authorization Application line extension for Orkambi in children ages 2 through 5 years has been submitted to the European Medicines Agency with a decision anticipated in the first half of 2019.