FDA Approves Treatment for Rare Enzyme Disorder
The FDA approved Mepsevii for treatment of children and adults with MPS VII, or Sly syndrome.
The syndrome is a rare condition affecting tissues and organs, affecting fewer than 150 individuals worldwide. Most patients have progressive skeletal abnormalities.
The approval was based on clinical trials involving 23 patients between five months and 25 years old. Common side effects of the drug include infusion site reactions and anaphylaxis.