June 27 2013 | By Márcio Barra
GlaxoSmithKline announced today that it has obtained Breakthrough Therapy designation from the FDA for it’s in-development medicine Drisapersen, a late-stage treatment for Duchenne muscular dystrophy, a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. Drisapersen is an antisense oligonucleotide, which induces exon skipping of exon 51 in cases where the child has dystrophin gene mutations due to an exon 51 skip.
The Breakthrough Therapy designation was based on results from the Phase II Study (DMD114117). The most prominent adverse event that occured during the trials was proteinuria – excess level of protein in the urine.
This is GlaxoSmithKline’s first Breakthrough Therapy designation from the FDA, since the program has enacted in 2012 as part of the Food and Drug Administration Safety and Innovation Act (FDASIA).
GlaxoSmithKline is developing drisapersen under an exclusive, worldwide license from the Dutch company, Prosensa.
Drisapersen closest competitor is Sarepta’s Eterplirsen drug, who showed impressive midtage data from a small trial. The company is currently trying to convince the FDA to grant an accelerated approval on eterplirsen.