FDA: New Option for Patients With Rare Metabolic Disorder

The US Food and Drug Administration approved Mepsevii (vestronidase alfa-vjbk) for the treatment adult and pediatric patients with mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome.

MPS VII is a rare disorder that affects less than 150 people worldwide and is characterized by a deficiency in the enzyme beta-glucuronidase. Mepsevii is an enzyme replacement therapy that works by replacing beta-glucuronidase.
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Approval for Mepsevii was based on a clinical trial and expanded access protocol that included 23 patients from 5 months to 25 years of age. Patients received 4mg/kg of Mepsevii once every 2 weeks for up to 164 weeks. Among the 10 patients who were able to complete the 6-minute walk test, the mean different in distance walked increased by 18 meters at 24 weeks and patients continued to show improvement or stabilization at 120 weeks. In addition, 2 patients who received Mepsevii experienced improvements in pulmonary function.

Common adverse effects associated with treatment included infusion site reactions, diarrhea, rash, and anaphylaxis.

—Melissa Weiss

Reference:

FDA approves treatment for rare genetic enzyme disorder [press release]. FDA. November 15, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585308.htm?utm_campaign=11152017_FDA%20approves%20treatment%20rare%20genetic%20enzyme%20disorder&utm_medium=email&utm_source=Eloqua. Accessed November 15, 2017.