FDA approves Xenpozyme, first treatment for rare acid sphingomyelinase deficiency
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The FDA has approved Xenpozyme as the first treatment for patients with acid sphingomyelinase deficiency with symptoms unrelated to the central nervous system.
Acid sphingomyelinase deficiency (ASMD) is a rare condition caused by the lack of an enzyme necessary to break down a complex lipid — sphingomyelin — which collects in the liver, spleen, lung and brain. Patients with ASMD often have enlarged abdomens that can cause pain, vomiting, feeding difficulties and increased risk for falls, as well as accompanying abnormal liver and blood tests.
Xenpozyme (olipudase alfa, Genzyme) is an enzyme replacement therapy that reduces sphingomyelin accumulation in the liver, spleen and lung. The FDA based its approval on results from a randomized, double-blind, placebo-controlled trial in which Xenpozyme improved lung function and reduced liver and spleen size among patients with ASMD.
“ASMD has a debilitating effect on people’s lives and there is a critical need to increase treatment options for patients who suffer from this rare disease,” Christine Nguyen, MD, deputy director for the office of rare diseases, pediatrics, urologic and reproductive medicine at the FDA, said in a press release. “The challenges involved with developing treatments for rare diseases are significant and unique. We believe patients who suffer from ASMD, their families and their physicians will welcome this long-awaited advancement.”
The most common adverse events associated with Xenpozyme included headache, cough, fever, joint paint, diarrhea and low blood pressure; however, the drug has a black box warning for severe hypersensitivity reactions, including anaphylaxis
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