The U.S. Food and Drug Administration (FDA) approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 6 to 11, who have two copies of the F508del mutation.
Drug Targets the Underlying Cause of the Disease in People with the Most Common CF Mutation
Today we received the exciting news that the U.S. Food and Drug Administration has approved Orkambi for people with cystic fibrosis ages 12 and older who have two copies of the F508del gene mutation, the most common CF mutation.
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.
CF Foundation Provided Scientific, Clinical and Significant Funding Support in Drug Development Process
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.