Vertex Cystic Fibrosis Treatment Gets US Approval for Use in 6- to 11-Year-Olds

By Reuters Staff

June 10, 2021

(Reuters) - Vertex Pharmaceuticals Inc said on Wednesday the U.S. Food and Drug Administration has approved the expanded use of its cocktail therapy, Trikafta, to treat cystic fibrosis in children aged between 6 to 11.

The drug Trikafta, approved in 2019 to treat patients 12 years and older, is expected to reach about 1,500 newly eligible children in the United States, the company said.

The treatment, currently priced at $311,503 per year, targets a defective protein responsible for the rare, life-threatening disease that builds up thick mucus in body parts such as the lungs and digestive tract.

Trikafta consists of backbone drugs ivacaftor and tezacaftor, and generated sales of $3.86 billion in 2020.

Vertex has submitted applications for use of the cocktail therapy in children aged 6 through 11 years to the European Medicines Agency and the Medicines & Healthcare Products Regulatory Agency.

The company also plans to file for the expanded use in Switzerland, Australia and Israel this year.