BioMarin Pharmaceutical Inc. has recently announced that the United States Food and Drug Administration (FDA) attributed a rare pediatric disease designation for drisapersen, a potential treatment for individuals suffering with Duchenne Muscular Dystrophy (DMD) who are responsive to exon 51 skipping treatment.
Drisapersen has been previously granted Orphan Drug and Fast Track Status designation, Breakthrough Therapy Designation and a Priority Review status, attributed to drugs that give major advances in treatment or that support a treatment for which no adequate therapy exist.
Duchenne muscular dystrophy is a type of recessive X-linked muscular dystrophy that strikes 1 in each 3,600 boys, resulting in muscle degeneration and, ultimately, premature death.
“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 U.S. 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,” noted Jean-Jacques Bienaimé, who is the Chairman and CEO of BioMarin.
According to the FDA a “rare pediatric disease” is as a condition that strikes less than 200,000 individuals in the United States with ages between birth and 18 years. As such, in the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor that earns an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease might be eligible for a voucher which can be redeemed to achieve priority review for a marketing application for a subsequent and different product. The Priority Review Voucher might be sold or transferred for an unlimited time.
BioMarin advances and commercializes innovative biopharmaceuticals to address preoccupying diseases and serious medical conditions. The firm’s product portfolio includes 5 approved products and several clinical and pre-clinical candidates. The approved products include Vimizim®, Naglazyme®, Aldurazyme®, Kuvan® and Firdapse®.