31. Trikafta

Vertex Pharma

Active ingredient: elexacaftor/ivacaftor/tezacaftor
Disease: cystic fibrosis
Peak sales estimate: $6.6 billion by 2025

Approved: Oct. 21, 2019

Company: Vertex

The scoop: With its five-months-early FDA approval for Trikafta, Vertex ushered in the triple combo era in cystic fibrosis—and majorly expanded its treatable population in the process. Cleared for CF patients over the age of 12 with either one or two copies of the delF508 CFTR gene mutation, the drug is eligible to treat 90% of U.S. cystic fibrosis patients, nearly doubling the 50% that Vertex drugs were already approved for. Analysts expect Trikafta, armed with a list price of $311,000, to shoot to blockbuster heights as early as 2020, though in doing so, it’ll hurt sales for the company’s older drugs. “Trikafta takes the efficacy benefit for CF patients to a whole new level compared to Symdeko and Orkambi, which are likely to fade away relatively quickly in all but the most stable patients (or those unable to access the triple due to geography or age),” SVB Leerink analyst Geoffrey Porges wrote at the time of approval. — Carly Helfand

31. Trikafta

Suggested Articles

Cancer biotech Zentalis Pharmaceuticals priced its IPO at $165 million, eclipsing the $100 million goal it laid out in early March.

The agreement gives Innovax access to a GSK adjuvant to enhance the immune response triggered by its recombinant protein-based vaccine.

Johnson & Johnson is paying Fate Therapeutics $50 million upfront to collaborate on up to four CAR NK and CAR-T cell therapies.