Who Owns Trikafta?

Trikafta is the fourth medicine in Vertex's cystic fibrosis franchise and the culmination of two decades of work on the CFTR protein. The sequence began with Kalydeco in 2012, the first drug to correct the underlying protein defect but effective for only about 4% of patients with specific mutations. Orkambi (2015) and Symdeko (2018) each combined two modulators and expanded coverage to the most common mutation, F508del, though with modest efficacy.

The triple combination added a third molecule, elexacaftor, a "corrector" that helps more defective CFTR protein reach the cell surface, on top of tezacaftor and the "potentiator" ivacaftor. In Phase 3 trials reported in 2019, the combination improved lung function by roughly 14 percentage points of predicted FEV1 and cut pulmonary exacerbations by 63%, results far beyond any prior CF therapy.

The FDA approved Trikafta on October 21, 2019, about five months after filing, one of the fastest reviews on record. Label expansions followed: children aged 6 to 11 in 2021, and ages 2 to 5 in 2023, extending the drug to patients as young as two years old. Europe approved the combination as Kaftrio in August 2020 after a public pricing dispute with the NHS.

Revenue scaled immediately. The product line passed $5.7 billion in 2021 and $8.9 billion in 2023 before reaching $10.3 billion in FY2025. In December 2024, Vertex won approval for Alyftrek, a once-daily successor designed to extend the franchise's patent life and broaden mutation coverage. Vertex expects most patients to transition to Alyftrek over time, which means Trikafta's revenue plateaued in 2025 even as the CF business kept growing.