Mepsevii Approved for Mucopolysaccharidosis VII
November 15, 2017 – The U.S. FDA has approved Mepsevii™ (vestronidase alfa-vjbk) injection, manufactured by Ultragenyx Pharmaceutical, Inc., to treat patients who have mucopolysaccharidosis type VII (MPS VII), also known as Sly syndrome. MPS VII is a genetic disorder that leads to shortages of the beta-glucuronidase enzyme in the body. This enzyme is needed to metabolize large sugar molecules called glycosaminoglycans (GAGs). Without it, GAGs accumulate in the body over time, leading to enlargement of tissues and organs. Signs and symptoms of MPS VII include bone deformities, developmental delays, organ damage, and respiratory difficulties. The disease is rare, affecting an estimated 1 in 250,000 births, but is typically fatal; the majority of patients do not reach adulthood.
Mepsevii acts as an enzyme replacement for beta-glucuronidase. Recommended dosing is based on weight. Patients should receive a four-hour intravenous infusion once every two weeks, at a dosage of 4mg/kg of body weight. A black box warning cautions that there is potential for severe allergic reactions. As a result, Mepsevii should be administered in a facility prepared to respond to an emergency, and patients should stay for monitoring for at least one hour after every infusion.
The manufacturer has already launched Mepsevii, which is available in single-use 10mg/5mL vials.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- U.S. Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-FDA-0178
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