Vertex Seeks US and European Approval for Combination Therapy to Treat Cystic Fibrosis

Vertex Pharmaceuticals has applied to the U.S. Food and Drug Administration (FDA) and  the European Medicines Agency (EMA) for approval of a new combination therapy for people 12 and older with cystic fibrosis, who have two copies of the most common genetic mutation associated with the disease, the F508del.

There are approximately 22,000 people with cystic fibrosis ages 12 and older who have two copies of the F508del mutation in North America, Europe and Australia, including approximately 8,500 in the United States and 12,000 in Europe. More than 1,000 mutations in the CFTR gene have been identified in people with cystic fibrosis.

For more information, visit the Rare Disease Report and Vertex press release.