Vertex’s CF Therapy Orkambi Receives Positive Recommendation for FDA Approval

After the Pulmonary-Allergy Drugs Advisory Committee (PADAC) met to discuss the approval of Orkambi, members of the committee voted 12 to 1 recommending Vertex Pharmaceuticals’ new treatment for cystic fibrosis to be approved by the US Food and Drug Administration for marketing. The indication is for cystic fibrosis patients who are at least 12 years old and have two copies of the F508del mutation.

“Today’s positive recommendation brings the cystic fibrosis community one step closer to potential approval of the first medicine to treat the underlying cause of this disease for many more people,” said Jeffrey Chodakewitz, MD, Executive Vice President and Chief Medical Officer of Vertex, in a news release from the company. “We look forward to continuing to work with the FDA and other regulatory agencies throughout the world to make Orkambi available to eligible patients as soon as possible.”

If officially approved by the FDA, Orkambi has the potential to serve a major portion of patients with cystic fibrosis. Approximately 8,500 cystic fibrosis patients in the United States are ages 12 years or older and are homozygous for the F508del mutation. This is out of approximately 75,000 individuals in the world living with cystic fibrosis.

As described in detail the day before the decision, Orkambi is a combination treatment of lumacaftor and ivacaftor. Together, the two drugs work to increase the amount of functional CFTR protein at the cell surface. A number of clinical trials evaluated combination treatment in patients with cystic fibrosis, and data from these trials were discussed by the PADAC.

The PADAC reviewed the safety and efficacy profiles from each trial to determine the acceptability of marketing Orkambi to cystic fibrosis patients. Members of the PADAC are experts in the fields of pulmonary medicine, allergy, clinical immunology, and epidemiology or statistics. By issuing a positive recommendation, the PADAC is highly influencing the FDA to approve Orkambi to treat cystic fibrosis in indicated patients.