Product approvals in regenerative medicine and rare diseases are among the highlights in our recap of recent FDA regulatory decisions.

According to Vertex Pharmaceuticals, approximately 300 additional people with CF are now eligible to receive treatment.

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Vertex has won two cystic fibrosis approvals from the FDA, but the agency included high-level safety warnings.

Alyftrek is approved as a new treatment for cystic fibrosis, while the FDA expanded the label for Trikafta to include additional genetic mutations.

The US Food and Drug Administration has approved Alyftrek, a once-daily oral combination of vanzacaftor, tezacaftor, and ...

Cystic fibrosis (CF) is an autosomal recessive disorder caused by mutations in the CF transmembrane regulator (CFTR) gene, leading to progressive lung disease and systemic complications. Lung disease ...

Alyftrek is a combination of three drugs — vanzacaftor (a CFTR potentiator), deutivacaftor (a CFTR corrector) and tezacaftor. We expect sales of this drug to drive growth in the medium term.

ALYFTREK ™ is approved for patients 6 years and older with at least one responsive mutation, including 31 additional mutations not responsive to other CFTR modulator therapies - - In head-to-head ...

Different genetic mutations cause different problems with this particular protein -- so CFTR modulators generally don't work ...