Research News

The U.S. Food and Drug Administration 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.

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.

La Administración de Alimentos y Medicamentos de los Estados Unidos ha aprobado el uso del modulador de combinación triple elexacaftor/tezacaftor/ivacaftor (Trikafta™) para las personas con fibrosis quística de 12 años en adelante que tienen al menos una copia de la mutación F508del.

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.