- Kalydeco was approved in the EU for use in cystic fibrosis (CF) patients with specific CF-related genetic mutations in July 2012.
- Kalydeco works by making certain defective proteins function more normally. Previously, only 4% of all CF patients were eligible -- a number that has now grown significantly, if incrementally.
- The expanded indication is based on phase III data in patients with a non-G551D gating mutation.
Last week, BioPharma Dive provided an in-depth look at the pricing controversy surrounding Vertex Pharma’s Kalydeco, which costs $307,000 per year in the U.S. Only about 4% of CF patients at the time were eligible for treatment with Kalydeco, which targets underlying genetic mutations -- but now researchers are investigating ways to expand the target treatment population.
Regulators in the EU have taken the step of expanding the target CF treatment population beyond patients with one of none specific CF transmembrane conductase (CF-TR) inhibitor mutations. A phase III study of 39 CF patients with a non-G551D gating mutation showed Kalydeco providing the intended benefits to the study population. During the study, patients experienced significant improvements in lung function, as well as other CF-related symptoms. Expanded use of this breakthrough drug -- even for just 250 additional patients -- is a very positive step forward.