FDA approves Ultragenyx's rare disease drug

Ultragenyx Pharmaceutical earned Food and Drug Administration approval for the ultra- rare disease drug Mepsevii.

Mepsevii is an enzyme replacement therapy for patients with Mucopolysaccharidosis VII, an inherited metabolic condition. The disease is extremely rare, affecting an estimated 200 patients globally.

"Prior to today's approval, patients with this rare, inherited condition had no approved treatment options," said Julie Beitz, MD, director of the Office of Drug Evaluation III in the FDA's Center for Drug Evaluation and Research.

Ultragenyx expects Mepsevii to hit the market later this month.

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