Today, Vertex Pharmaceuticals Inc. announced that the U.S. Food and Drug Administration has accepted its application to expand Trikafta® (elexacaftor/tezacaftor/ivacaftor) to include children ages 6-11 years old with cystic fibrosis who have at least one F508del or a mutation in the CFTR gene that is responsive based on in vitro data. The FDA has granted priority review of the application and has indicated that it will make a decision by June 8, 2021.
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.
Roche has acquired a set of potentiator compounds from Enterprise Therapeutics and intends to study them for the treatment of CF. The Cystic Fibrosis Foundation previously provided funding to Enterprise to develop these potential medicines.
Thirty-three new projects are being funded as part of the Foundation's $100 million Infection Research Initiative.
The Cystic Fibrosis Foundation has awarded up to $5.6 million to Microbion Corporation to develop a novel, inhaled antibiotic to treat drug-resistant bacterial infections in people with cystic fibrosis.
The Cystic Fibrosis Foundation awarded up to $5 million to Armata Pharmaceuticals for the first-ever controlled clinical study of phage therapy in CF, reaffirming the Foundation's commitment to advance innovative solutions to the growing challenge of antibiotic resistance.
The Cystic Fibrosis Foundation has entered into an agreement with Synspira Therapeutics Inc. to develop a non-porcine enzyme replacement therapy to offer an alternative to people with cystic fibrosis who cannot digest food properly.
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.
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.