Also known as: Kaftrio, KAFTRIO
Vertex Pharmaceuticals dominates cystic fibrosis therapies with TRIKAFTA/KAFTRIO generating $2.57 billion in Q4 2025 and ALYFTREK ramping to $380.1 million. The company posted strong free cash flow of $3.19 billion in 2025 and is expanding into pain, kidney disease, and gene-editing therapies.
Biotech investors are closely watching cystic fibrosis drug trials, rare-disease therapies and regulatory catalysts. Sionna Therapeutics is advancing a new CF drug class, while Praxis Precision Medicines targets essential tremor. FDA staffing challenges add uncertainty to approval timelines.
Vertex Pharmaceuticals and CRISPR Therapeutics reported $116 million in full-year 2025 sales for gene-editing therapy Casgevy, with 64 patients receiving infusions and patient initiations nearly tripling compared to 2024.
