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news.The US Food and Drug Administration (FDA) has granted rare pediatric disease designation for BioMarin Pharmaceutical's drisapersen, a potential treatment for patients with Duchenne Muscular Dystrophy (DMD) who are amenable to exon 51 skipping treatment.
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Earlier, drisapersen has also secured orphan and fast track status, breakthrough therapy designation, as well as priority review status from the FDA.
According to FDA, a rare pediatric disease affects fewer than 200,000 individuals in the US manily aged from birth to 18 years.
BioMarin chairman and chief executive officer Jean-Jacques Bienaimé said: "BioMarin has a track record of efficiently developing therapies to treat rare and ultra-rare genetic diseases and we are committed to bringing drisapersen to children who desperately need an approved treatment option in the US.
"We are pleased that the FDA’s Office of Orphan Products Development has granted drisapersen this designation.
"BioMarin was the first Company to receive a Rare Pediatric Disease Priority Review Voucher upon the approval of Vimizim, in February of 2014.
"We are dedicated to developing important new therapeutics for patients, mostly children, with life threatening disorders, and this designation for drisapersen is a testament to BioMarin’s deep commitment to that goal."
The company is focused on developing and commercializing new biopharmaceuticals for serious diseases and medical conditions.
Its product portfolio has five approved products including Vimizim, Naglazyme, Aldurazyme, Kuvan, Firdapse.