FDA approves Orkambi (lumacaftor + ivacaftor) to treat cystic fibrosis F508del mutation- Vertex Pharma

The FDA has approved Orkambi (lumacaftor + ivacaftor) combination from Vertex Pharma for the treatment of cystic fibrosis patients whose condition is caused by the F508del mutation in their CFTR genes. This decision opens a larger market for Vertex Pharma whose Kalydeco (lumacaftor) was approved as a monotherapy for cystic fibrosis with different CFTR mutations.

The approval of Orkambi was based on data from two Phase III studies (TRAFFIC and TRANSPORT) that enrolled more than 1,100 people with cystic fibrosis ages 12 and older with two copies of the F508del mutation. Patients treated with Orkambi experienced statistically significant improvements in lung function. Patients also experienced reductions in pulmonary exacerbations and improvements in body mass index (BMI). The most common adverse events included shortness of breath and/or chest tightness, upper respiratory tract infection (common cold) and gastrointestinal symptoms (including nausea, diarrhea, or gas).

Comment: Orkambi will be the first and only medicine to treat the underlying cause of cystic fibrosis for eligible people ages 12 and older with two copies of the F508del mutation in the CFTR gene. People with two copies of the F508del mutation represent the largest group of people with cystic fibrosis. There are approximately 8,500 people ages 12 and older with two copies of the F508del mutation in the USA.