FDA Approves Vertex's Cystic Fibrosis Drug Orkambi

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) said Thursday that the U.S. Food and Drug Administration has approved Orkambi, the first medicine to treat the underlying cause of cystic fibrosis in people ages 12 and older with two copies of the F508del mutation.

Cystic fibrosis is a rare, life-threatening genetic disease. People with two copies of the F508del mutation represent the largest group of people with cystic fibrosis. Of the 30,000 people in the United States with cystic fibrosis, about 8,500 ages 12 and older have two copies of the F508del mutation.

Vertex said Orkambi will be available for shipment to specialty pharmacies in the United States within days.

The approval of Orkambi was based on data from two Phase 3 studies 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.

Orkambi is a combination of lumacaftor, which is designed to increase the amount of mature protein at the cell surface by targeting the processing and trafficking defect of the F508del CFTR protein, and ivacaftor, which is designed to enhance the function of the CFTR protein once it reaches the cell surface. The drug is taken every 12 hours - once in the morning and once in the evening.

Vertex shares are currently trading at $130.95, up $4.76 or 3.77%.