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FDA Alerts
Genetic and Congenital

FDA approves olipudase alfa for acid sphingomyelinase deficiency

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The U.S. Food and Drug Administration approved olipudase alfa (Xenpozyme) for intravenous infusion in patients with non-central nervous system (non-CNS) manifestations of Acid Sphingomyelinase Deficiency (ASMD), a rare genetic disease.

This is the first disease-specific treatment approved for ASMD (non-CNS manifestations).

The most common side effects include headache, cough, fever, joint pain, diarrhea, and low blood pressure.

“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,” said Christine Nguyen, MD, deputy director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine in the FDA’s Center for Drug Evaluation and Research 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.”

Read the full press release here.

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