Ultragenyx announces FDA approval of MEPSEVII
Ultragenyx Pharmaceutical (RARE) announced that the U.S. Food and Drug Administration has approved MEPSEVII, the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis VII.
MEPSEVII is an enzyme replacement therapy designed to replace the deficient lysosomal enzyme beta-glucuronidase in MPS VII patients.
MPS VII is a mucopolysaccharide disease also known as Sly syndrome.
MPS VII is a rare genetic, metabolic lysosomal storage disorder caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans dermatan sulfate, chondroitin sulfate and heparan sulfate. These complex GAG carbohydrates are a critical component of many tissues.
The inability to properly break down GAGs leads to a progressive accumulation in many tissues and results in a multi-system tissue and organ damage.
MPS VII is one of the rarest MPS disorders, with an estimated 200 patients in the developed world.
MEPSEVII was evaluated by the FDA with Priority Review, which is reserved for drugs that offer major advances in treatment or provide a treatment where no adequate therapy exists.
With this approval, the FDA issued a Rare Pediatric Disease Priority Review Voucher, which confers priority review to a subsequent drug application that would not otherwise qualify for priority review.
The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.
MEPSEVII will be available to patients in the U.S. later this month.
In Europe, the European Medicines Agency is currently reviewing the Marketing Authorization Application for vestronidase alfa, and an opinion from the Committee for Medicinal Products for Human Use is expected in the first half of 2018.
RARE last traded at $46.44, up 88 cents.
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