ULTRAGENYX ANNOUNCES FDA APPROVAL OF MEPSEVII[TM]: Mepsevii (Vestronidase Alfa) Is the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis Vii

The Exceptional Parent, December 2017 | Go to article overview

ULTRAGENYX ANNOUNCES FDA APPROVAL OF MEPSEVII[TM]: Mepsevii (Vestronidase Alfa) Is the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis Vii


Ultragenyx Pharmaceutical Inc., a biopharmaceutical company focused on the development of novel products for rare and ultra-rare diseases, recently announced that the U.S. Food and Drug Administration (FDA) has approved MEPSEVII[TM] (vestronidase alfa), the first medicine approved for the treatment of children and adults with Mucopolysaccharidosis VII (MPS VII, Sly syndrome). MEPSEVII is an enzyme replacement therapy designed to replace the deficient lysosomal enzyme beta-glucuronidase in MPS VII patients.

"The approval of MEPSEVII is a pivotal moment for Ultragenyx and for patients suffering from ultrarare genetic diseases for which the investment and development of treatments has not happened yet," said Emil D. Kakkis, M.D., Ph.D., Chief Executive Officer and President of Ultragenyx. "Our development program sought to create a new paradigm in study design and endpoint evaluations to help accommodate the difficulties of studying extremely heterogeneous ultra-rare diseases to fulfill the promise that the science we have all invested in over many years actually becomes something available for patients. I would like to thank the patients and their families for their participation in the clinical program."

MPS VII is a rare genetic, metabolic lysosomal storage disorder (LSD) caused by the deficiency of beta-glucuronidase, an enzyme required for the breakdown of the glycosaminoglycans (GAGs) 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.

"I am thrilled beyond belief to see this treatment advance after more than 40 years of work and anticipation. Thanks to Ultragenyx for making it happen," said William S. Sly, Chairman Emeritus, Department of Biochemistry at St. Louis University. "I hope that this treatment will follow the other successful examples of enzyme therapy for LSDs and help improve the lives of patients with this rare disease."

MEPSEVII[TM] (vestronidase alfa-vjbk) 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 in November. In order to support patients, Ultragenyx has launched UltraCare[TM], a comprehensive support service that will provide ongoing support to patients and caregivers. …

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ULTRAGENYX ANNOUNCES FDA APPROVAL OF MEPSEVII[TM]: Mepsevii (Vestronidase Alfa) Is the First Therapy for Progressive and Debilitating Rare Genetic Disease Mucopolysaccharidosis Vii
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