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29 Oct 2019

Insighht on USFDA approval of Trikafta

Vertex Pharmaceuticals received approval from the U.S. Food and Drug Administration (FDA).Trikafta

This is a landmark approval where Vertex’s Trikafta earned this approval only three months after the application was filed, five months sooner than actually anticipated.

Trikafta is a triple-drug combination (elexacaftor/ivacaftor/tezacaftor) that targets the genetic causes of cystic fibrosis in patients. Specifically, it is designed for patients who have a particular gene mutation that leads to the symptoms of the disease. This mutation, known as F508del, is estimated to be present in around 90% of the cystic fibrosis patient population.

Trikafta is a triple-drug combination that targets the genetic causes of cystic fibrosis in patients. Specifically, it is designed for patients who have a particular gene mutation that leads to the symptoms of the disease. This mutation, known as F508del, is estimated to be present in around 90% of the cystic fibrosis patient population.

The FDA granted this application Priority Review, in addition to Fast Track and Breakthrough Therapy Designation. Trikafta also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. Drugs approved under expedited programs are held to the same approval standards as other FDA approvals. Because of Trikafta’s benefit to the cystic fibrosis community, the FDA reviewed and approved Trikafta in approximately three months, ahead of the March 19, 2020 review goal date. The approval of Trikafta was granted to Vertex Pharmaceuticals Incorporated, which will receive a Rare Pediatric Disease Priority Review Voucher for developing this therapy.

 

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