Drug Now FDA-Approved In U.S. To Treat Rare Enzyme Disorder

Alexion Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration has approved Kanuma (sebelipase alfa) for patients of all ages diagnosed with a very rare disease known as lysosomal acid lipase deficiency (LAL-D).

This incredibly rare disease occurs when the body does not produce enough active lysosomal acid lipase (LAL or LIPA) enzyme, which is essential for breaking down fatty material in the body. For those suffering with from this health issue, they may deal with a build-up of fatty material in a number of their organs, including the gut, liver, spleen and wall of the blood vessels. In turn, this can lead to liver failure, heart disease and early death. 

"We are pleased with the FDA approval of Kanuma, a transformative treatment for patients with LAL-D, a devastating, ultra-rare disease that causes premature death in infants and multi-organ damage in those who survive," said David Hallal, Chief Executive Officer of Alexion. "Importantly, the label includes a survival benefit in infants and reductions in important markers of liver disease, including ALT and liver fat content, as well as significant improvements in lipid parameters, in children and adults. This approval also strengthens Alexion's global leadership in rare diseases as we broaden our product portfolio to transform the lives of more patients with severe and life-threatening disorders. We look forward to bringing Kanuma to patients with LAL-D and their physicians in the United States."

Data based on Kanuma's approval in the United States comes from two clinical studies and a supporting open-label extension study involving adult patients and pediatric patients with the health issue, according to a news release. 

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