FDA Approves Genzyme's Xenpozyme For Rare Genetic Disease

(RTTNews) - The U.S. Food and Drug Administration approved Genzyme's Xenpozyme (Olipudase alfa) for intravenous infusion in pediatric and adult patients with Acid Sphingomyelinase Deficiency or ASMD, a rare genetic disease that causes premature death.

Xenpozyme is the first approved medication to treat symptoms that are not related to the central nervous system in patients with ASMD.

Acid Sphingomyelinase Deficiency is caused by the lack of an enzyme needed to break down a complex lipid, called sphingomyelin, that accumulates in the liver, spleen, lung, and brain. Patients with ASMD have enlarged abdomens that can cause pain, vomiting, feeding difficulties, and falls. They also have abnormal liver and blood tests. The most severely affected patients have profound neurologic symptoms and rarely survive beyond two to three years of age. Other patients may survive into adulthood but die prematurely from respiratory failure.

Xenpozyme is an enzyme replacement therapy that helps reduce sphingomyelin accumulation in the liver, spleen, and lung.

The most common side effects of Xenpozyme include headache, cough, fever, joint pain, diarrhea, and low blood pressure. Xenpozyme carries a boxed warning for severe hypersensitivity reactions including anaphylaxis.

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