With the announcement today, Kalydeco is now approved for people with the following mutations: G551D, G178R, S549N, S549R, G551S, G1244E, S1251N, S1255P and G1349D. In 2012, the
In the United States, approximately 150 people have one of the additional eight mutations for which Kalydeco is now approved. Kalydeco, a breakthrough therapy, is the first drug to treat the underlying cause of cystic fibrosis.
“Kalydeco's expanded approval is welcome news for the CF community,” said Robert J. Beall, Ph.D., president and CEO of the Cystic Fibrosis Foundation. “We continue to support research with Kalydeco in more patient groups, and at the same time, are aggressively pursuing the development of other promising compounds that treat the root cause of CF and benefit the greatest number of people with cystic fibrosis.”
In the group of eight mutations, and G551D, the defective
The FDA expanded Kalydeco's use based on results of a Phase 3
Data from the study did not support approval of the drug in patients with the G970R mutation. Vertex estimates that about 10 people worldwide have this mutation, including two in this country.
Kalydeco was developed by Vertex Pharmaceuticals, with a $75 million investment from the CF Foundation, which was used to discover this drug and other potential treatments still in development.
The Foundation is investing in a robust pipeline of potential therapies that target the disease from every angle, including research collaborations with Pfizer, Genzyme and Vertex to accelerate the discovery and development of new drugs.