Posted by Michael Wonder on 20 Aug 2018
Wave Life Sciences receives US orphan drug and rare paediatric disease designations for WVE-210201
16 August 2018 - Wave Life Sciences today announced that the U.S. FDA has granted both orphan drug designation and rare pediatric disease designation for WVE-210201 for the treatment of Duchenne muscular dystrophy.
The European Commission previously granted orphan drug designation for WVE-210201 in July 2018.
WVE-210201 is currently being studied in an ongoing global, multicenter, double-blind, placebo-controlled Phase 1 clinical trial designed to evaluate the safety, tolerability and plasma concentrations of single ascending doses of WVE-210201 administered intravenously in Duchenne muscular dystrophy patients with gene mutations amenable to exon 51 skipping. The trial is expected to enroll up to 40 patients, including ambulatory and non-ambulatory patients, between the ages of 5 and 18 years of age. As patients complete the Phase 1 trial, they have the option to enrol in an ongoing open label extension study in which they receive continued treatment with WVE-210201.
