Nonprofit issues challenge to accelerate treatments for every person with CF
The Cystic Fibrosis Foundation announced today that it has licensed a compound to the biopharmaceutical company AbbVie to develop into a potential CFTR modulator treatment.
The U.S. Food and Drug Administration has approved the use of the triple-combination modulator elexacaftor/tezacaftor/ivacaftor (Trikafta™) for people with cystic fibrosis ages 12 and older who have at least one copy of the F508del mutation.
This medicine represents the single greatest therapeutic advancement in the history of CF, offering a treatment for the underlying cause of the disease that could eventually benefit more than 90 percent of people with CF.
The Cystic Fibrosis Foundation has awarded up to $7 million to Enterprise Therapeutics to develop a compound that targets a non-CFTR chloride channel in lung cells. If successful, the drug could help mucus become more hydrated and easier to clear from the lungs of all people with CF, regardless of their CFTR mutations.
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 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.