Elexacaftor/ivacaftor/tezacaftor

New real-world evidence presented at ATS 2025 validates Trikafta (elexacaftor/tezacaftor/ivacaftor) as an effective long-term therapy for cystic fibrosis patients with at least one F508del mutation.

Royalty Pharma shareholders have overwhelmingly approved the acquisition of its external manager, with 99.9% voting in favor at the company's 2025 Annual General Meeting.

Vertex Pharmaceuticals reported Q1 2025 revenue of $2.77 billion, representing a 3% year-over-year increase, driven by successful launches of ALYFTREK and CASGEVY.

Orphan drug sales reached $168 billion globally in 2023, with growth expected to moderate to just under 10% annually through 2028, according to Evaluate's latest report.

NIH's substantial investment of $84 million annually in cystic fibrosis research has led to groundbreaking developments in understanding and treating the disease.