Posted by Michael Wonder on 21 Jun 2019
FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older
21 June 2019 - The U.S. FDA today expanded the indication for Symdeko (a combination of tezacaftor/ivacaftor) tablets for treatment of paediatric patients ages 6 years and older with cystic fibrosis who have certain genetic mutations.
Last year, the FDA approved Symdeko to treat patients ages 12 and older who had the same specific genetic mutations.
The efficacy of Symdeko in patients with cystic fibrosis age 12 years and older was evaluated in three Phase 3, double blind, placebo-controlled trials, which demonstrated improvements in lung function and other key measures of the disease, including a reduction in exacerbations (a worsening of symptoms requiring treatment). The efficacy in patients ages 6 to 12 was extrapolated from patients age 12 years and older, with additional support from data in patients age 6 to 12 years.
