The U.S. Food and Drug Administration (FDA) announced today that it has approved the use of ivacaftor (Kalydeco™) to treat people with cystic fibrosis ages 6 and older who have the R117H mutation.
Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the Cystic Fibrosis Foundation, announced today a $15 million research agreement with biopharmaceutical company Shire plc to support the development of a new cystic fibrosis treatment targeting the underlying cause of the disease.
The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation's royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. The Foundation received $3.3 billion from the sale to Royalty Pharma.
CF Foundation President and CEO Robert J. Beall, Ph.D. testified today before the House of Representatives' Energy and Commerce Committee at a hearing entitled, “21st Century Cures: Incorporating the Patient Perspective.”
This morning, Vertex Pharmaceuticals announced the results from the Phase 3 clinical trials of ivacaftor (Kalydeco™) and lumacaftor (VX-809) in people with two copies of the F508del mutation.
Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), an affiliate of the Cystic Fibrosis Foundation, announced it will extend funding for continued collaboration with Proteostasis Therapeutics, Inc., for the development of new therapies to treat the most common CF mutation, Delta F508.
The U.S. Food and Drug Administration announced today it has approved Kalydeco™ to treat people ages 6 and older who have one of eight additional cystic fibrosis mutations.