News

The Cystic Fibrosis Foundation recently signed on to a letter with more than 70 organizations in opposition to the Right to Try Act, which passed in the House of Representatives earlier this month.

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.

Tezacaftor/ivacaftor (Symdeko™) is approved for individuals with two copies of the most common cystic fibrosis mutation, F508del, as well as for individuals who have a single copy of one of 26 specified mutations -- regardless of their other mutation.

Today Vertex Pharmaceuticals Inc. announced the two next-generation CFTR modulator candidates that will progress into Phase 3 clinical trials.

The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.

The U.S. Food and Drug Administration (FDA) today approved ivacaftor (Kalydeco^®) for people ages 2 and older who have at least one of 23 residual function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Corbus Pharmaceuticals reported promising results Thursday from an early stage clinical study of a potential anti-inflammatory drug for people with cystic fibrosis.

The Cystic Fibrosis Foundation today announced an unprecedented acceleration and expansion of its research, care and patient programs for the CF community. This action is possible as a result of the sale of the Foundation's royalty rights to CF treatments developed by Vertex Pharmaceuticals Inc. The Foundation received $3.3 billion from the sale to Royalty Pharma.

CF Foundation President and CEO Robert J. Beall, Ph.D. testified today before the House of Representatives' Energy and Commerce Committee at a hearing entitled, “21st Century Cures: Incorporating the Patient Perspective.”