Vertex Pharmaceuticals Nabs Key FDA Approval in Cystic Fibrosis


The Food and Drug Administration has approved Vertex Pharmaceuticals’ (NASDAQ:VRTX) supplemental application for Trikafta’s use in eligible cystic fibrosis patients aged 6 to 11 under priority review. The decision opens up access to Trikafta for the first time to an estimated 1,500 children with at least one F508del mutation.

Previously, Trikafta — a combination drug comprising the company’s elexacaftor, Symdeko, and Kalydeco — was only approved for use in amenable patients 12 and older.

Three healthcare professionals giving a thumb's up.

IMAGE SOURCE: GETTY IMAGES.

Vertex Pharmaceuticals is the market share leader in cystic fibrosis treatment; nearly 50% of patients currently being treated for the disease are receiving one of its medications. In 2020, Trikafta accounted for 63% of Vertex Pharmaceuticals’ $6.2 billion in product revenue, and in the first quarter of 2021, it represented 70% of the company’s $1.7 billion in sales.

Of the 83,000 cystic fibrosis patients in the U.S., Europe, Australia, and Canada, an estimated 30,000 are untreated, but could be amenable to Trikafta, according to the company. To make Trikafta more widely available, Vertex Pharmaceuticals is seeking expanded approvals and reimbursement agreements internationally. Currently, it’s available in 12 countries outside the U.S., including Germany, Ireland, Switzerland, and the United Kingdom.

The company is also conducting additional trials to prove Trikafta’s efficacy in more patients. For example, a clinical study evaluating its use in patients ages 2 to 5 is ongoing, and a trial involving patients with rare genetic mutations is planned. 

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