The Food and Drug Administration has given approval to Vertex Pharmaceuticals‘ cystic fibrosis drug Orkambi. The combination therapy is the first for CF aimed at treating the cause of the disease in people who have two copies of the F508del mutation, the most common genetic mutation behind cystic fibrosis.
Orkambi, a combination of Vertex’s approved Kalydeco and a new medication called lumacaftor, unlike current drugs which treat related symptoms, targets the underlying condition caused by the mutated gene.
The F508del mutation variation of cystic fibrosis affects about 8,500 people in the U.S. 12 years and older. The approval was announced on the agency’s website Thursday.
The wholesale cost of Orkambi is $259,000 per patient per year, less than the $311,000 annual bill for Kalydeco alone. That price has provoked criticism from some cystic fibrosis doctors.
In the Journal of the American Medical Association, the authors of the commentary write:
“Rare genetic diseases such as CF are increasingly seen as unique niche markets for pharmaceutical companies. An unsustainable price structure for novel drugs developed by pharmaceutical companies for this rare condition and others is developing. In January 2012, the US Food and Drug Administration approved Vertex Pharmaceuticals’ drug ivacaftor (Kalydeco) for use in a subpopulation of patients with CF who carry a specific genetic mutation, G551D. Ivacaftor is the first drug that treats the underlying molecular defect in CF, and its introduction illustrates an application of personalized medicine. The development program that led to the discovery of this drug was firmly grounded and incrementally built on the many contributions made during at least 3 decades by a highly supportive community built by and around patients and their families affected by this disease.”
The new drug, Boston based Vertex said, will be available within days.
Cystic fibrosis is a genetic disorder affecting mostly the lungs but also the pancreas, liver, kidneys and intestine. Long-term issues include difficulty breathing and coughing up sputum as a result of frequent lung infections. Death from respiratory failure occurs frequently when people are in their 20s, 30s or 40s.