Shots:   

• The US FDA has approved Alyftrek (QD, CFTR modulator) to treat patients (≥6yrs.) with cystic fibrosis (CF), having at least one F508del mutation or another CFTR gene mutation. Submissions are under review in the EU, UK, Canada, Switzerland, Australia & New Zealand 

• Approval was supported by pivotal P-III CF program involving >1,000 patients across 20+ countries, with results reported as conclusion of the studies & highlighted at the North American Cystic Fibrosis Conference in Sep 2024 

• P-III studies in CF patients (≥12yrs.) showed that Alyftrek met its 1EP of ppFEV1 non-inferiority & 2EP of SwCl improvement vs Trikafta. In children (6–11yrs.), the drug demonstrated safety (1EP) as well as benefits in ppFEV1 & SwCl  

Ref: Orna Therapeutics | Image: Orna Therapeutics & Vertex Pharmaceuticals

Related News: Vertex Receives CHMP’s Positive Opinion for Kalydeco as a Treatment of Cystic Fibrosis in Infants

PharmaShots! Your go-to media platform for customized news ranging for multiple indications. For more information connect with us at connect@pharmashots.com