News

The Cystic Fibrosis Foundation awarded up to $1.61 million to Eloxx Pharmaceuticals Inc. to conduct a U.S.-based Phase 2 clinical trial of an investigational drug that could potentially treat people with cystic fibrosis who have a nonsense mutation.

The U.S. Food and Drug Administration has approved the use of tezacaftor/ivacaftor (Symdeko®) for children with cystic fibrosis ages 6 to 11 with specific mutations.

The Institute for Clinical and Economic Review (ICER) is developing an assessment of the clinical effectiveness and value of the triple combination (also known as elexacaftor/tezacaftor/ivacaftor). The first opportunity to comment runs through Sept. 25.

The Cystic Fibrosis Foundation has increased its award to Arcturus Therapeutics to up to $15 million to develop a mutation-agnostic therapy for people with cystic fibrosis.

Vertex Pharmaceuticals Inc. announced today that it submitted a New Drug Application to the U.S. Food and Drug Administration for the approval of the first triple-combination therapy for cystic fibrosis.

Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).

The U.S. Food and Drug Administration has approved the use of ivacaftor (Kalydeco®) for children as young as 6 months.

The Cystic Fibrosis Foundation awarded up to $5.1 million to TB Alliance to advance a drug discovery program in partnership with Johns Hopkins University. The effort will identify compounds with the potential to treat nontuberculous mycobacteria infections that are increasingly found among people with CF.

Today, Vertex released positive preliminary clinical trial results for the potential triple-combination therapy VX-445 plus tezacaftor/ivacaftor (Symdeko®).

Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.