Trikafta is a co-packaged cystic fibrosis transmembrane conductance regulator modulator regimen combining elexacaftor, tezacaftor, and ivacaftor. It is indicated for cystic fibrosis in patients aged 2 years and older with at least one F508del mutation or other in vitro responsive CFTR mutations. U.S. initial approval was in 2019.
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.
Global orphan drug sales are forecast to reach $409 billion by 2032, representing one-fifth of prescription drug sales, with Johnson & Johnson and Argenx leading the market amid regulatory uncertainty.
Switzerland-based CRISPR Therapeutics stands to benefit from increased Casgevy sales following partner Vertex Pharmaceuticals' fourth-quarter results, with five additional gene-editing therapies in clinical trials targeting larger patient populations.
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.
Vertex Pharmaceuticals reported fourth quarter 2025 revenue of $3.19 billion, up 10% year-over-year, and provided full year 2026 revenue guidance of $12.95 billion to $13.1 billion with over $500 million expected from non-CF products.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05743946 |
Trikafta for Patients With Non-cystic Fibrosis Bronchiectasis |
COMPLETED | PHASE4 |
| NCT05279040 |
Trikafta Exercise Study in Cystic Fibrosis |
RECRUITING | |
| NCT05200429 |
Canadian Observational Study Evaluating the Long-term IMPACT of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Modulators on People With CF |
RECRUITING | |
| NCT03506061 |
Trikafta in Cystic Fibrosis Patients |
COMPLETED | PHASE2 |
