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.
Site Search
Leaders of the Cystic Fibrosis Foundation and dedicated volunteers from the CF community came together last week around a shared commitment to not stop working until a cure is found for every person living with cystic fibrosis.
The U.S. Food and Drug Administration (FDA) today expanded its approval of three CFTR modulators to include additional people with CF who have certain rare mutations. The approval enables more than 600 individuals with CF who were not previously eligible for modulators to access drugs that treat the underlying cause of their disease for the first time.
Today, the Cystic Fibrosis Foundation announced that it will invest up to $8.4 million in SpliSense's Series B funding round to develop an antisense oligonucleotide therapy for people with cystic fibrosis who have splicing mutations and potentially other rare mutations.
Foundation to fund up to $110 million for collaboration to accelerate the development of genetic therapies for CF
Phase 2 clinical trial results were announced today for a potential next-generation modulator to treat people with two copies of the most common cystic fibrosis mutation, F508del.
Vertex Pharmaceuticals Inc. today announced that it will pursue FDA approval for the triple-combination therapy VX-445 (elexacaftor) plus tezacaftor/ivacaftor (Symdeko®).
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 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.
Today, Vertex released positive preliminary clinical trial results for the potential triple-combination therapy VX-445 plus tezacaftor/ivacaftor (Symdeko®).