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.

Multiple CDMOs secured significant contract manufacturing opportunities as regulatory bodies approved new indications for established drugs, particularly in oncology and rare diseases.

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.