Vertex Pharmaceuticals Inc. priced Trikafta — its triple combination treatment for a type of cystic fibrosis — at $311,503 annually, following the drug's U.S. approval, Reuters reported.
Cystic fibrosis is a rare, life-threatening inherited disorder characterized by buildup of mucus in the lungs and digestive system — causing severe damage to the affected organs. The disease is due to changes in the CFTR gene, with F508del mutation being the most common gene defect.
After a three-month review period, the U.S. Food and Drug Administration on Oct. 21 approved Trikafta, a combination of elexacaftor, tezacaftor and ivacaftor, for patients with cystic fibrosis aged 12 and older who have at least one F508del gene mutation. The group represents 90% of cystic fibrosis patients.
The approval was based on results from two studies that showed Trikafta improved lung function among patients. The first study tested the drug combination in 403 patients with one F508del mutation and one minimal function mutation for 24 weeks, while the second evaluated 107 patients with two identical F508del mutations.
Trikafta was granted a number of designations that provided for expedited review and certain development perks, including priority review, fast track, breakthrough and orphan drug.
Leerink analysts estimate the therapy to hit the shelves by the end of the first quarter of 2020. Leerink also estimates sales of Trikafta to grow to about $4.6 billion in 2023 and $6.6 billion by 2025.
Boston-based Vertex Pharmaceuticals markets ivacaftor as Kalydeco and the combination of tezacaftor and ivacaftor as Symdeko.
Trikafta is being further examined in a phase 3 clinical trial in younger patients, with a goal to earn an approval for patients aged six through 11.