FDA Approves Genzyme’s Xenpozyme for ASMD

The FDA has approved Genzyme’s Xenpozyme (Olipudase alfa) intravenous infusion treatment for adult and pediatric patients with acid sphingomyelinase deficiency (ASMD), a rare genetic disease that causes premature death.

Xenpozyme is an enzyme replacement therapy designed to replace deficient or defective acid sphingomyelinase. ASMD is caused by the lack of the enzyme, which is needed to break down a complex lipid called sphingomyelin that accumulates in the liver, spleen, lung and brain.

The disease leads to enlarged abdomens that can cause pain, vomiting, feeding difficulties and falls, but also abnormal liver and blood tests, neurologic symptoms and respiratory failure.

In clinical trials, adult and pediatric patients saw improvements in lung function and reduced spleen and liver volumes. 

Xenpozyme is the first drug approved for treatment of this condition.