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Sly Syndrome Treatment OK'd (FDA)

— Enzyme replacement for rare liposomal storage disorder

MedpageToday

The , a.k.a. mucopolysaccharidosis type VII or MPS VII, the agency said Wednesday.

MPS VII is a lysosomal storage disorder in which beta-glucuronidase is deficient. The novel enzyme therapy, to be sold by Ultragenyx Pharmaceutical, is a recombinant version of the missing enzyme.

Data supporting the approval came from a clinical trial and also individual patients receiving the product through the FDA's expanded access program -- 23 patients in all. Patients receiving the agent showed an 18-meter improvement in 6-minute walk distance after 24 weeks relative to a placebo group. Longer treatment resulted in additional gains or stabilization.

"Overall, the results observed would not have been anticipated in the absence of treatment," the FDA said.