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news.Ultragenyx Pharmaceutical’s enzyme replacement therapy Mepsevii (vestronidase alfa) has secured approval from the US Food and Drug Administration (FDA) for the treatment of Mucopolysaccharidosis VII (MPS VII, Sly syndrome) in both children and adults.
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MPS VII is a progressive and debilitating inherited, rare genetic disease which previously didn’t have any approved therapy. It is a metabolic lysosomal storage disorder (LSD) resulting from the deficiency of beta-glucuronidase.
Mepsevii works by replacing the deficient lysosomal enzyme beta-glucuronidase in MPS VII patients.
Beta-glucuronidase is needed by the body for the breakdown of dermatan sulfate, chondroitin sulfate and heparan sulfate glycosaminoglycans (GAGs). These complex carbohydrates are a critical component of several tissues.
According to the FDA, less than 150 patients across the world are affected by MPS VII.
Ultragenyx CEO and President Emil Kakkis said: “The approval of Mepsevii is a pivotal moment for Ultragenyx and for patients suffering from ultra-rare genetic diseases for which the investment and development of treatments has not happened yet.
“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.”
The FDA stated that Mepsevii’s safety and efficacy was demonstrated in clinical trial and expanded access protocols in 23 patients, aged between 5 months to 25 years.
Ultragenyx has been asked to hold a post-marketing study for the enzyme replacement therapy to assess its long-term safety.
The California-based biopharmaceutical company plans to make Mepsevii available in the US later this month.
In Europe, vestronidase alfa is being reviewed by the European Medicines Agency (EMA), with an opinion from the Committee for Medicinal Products for Human Use (CHMP) anticipated to come in the first half of 2018.
Image: Mepsevii becomes the first FDA approved treatment for pediatric and adult patients with MPS VII. Photo: courtesy of The U.S. Food and Drug Administration/Commons.wikimedia.org.