Health & Medicine
Drug Now FDA-Approved In U.S. To Treat Rare Enzyme Disorder
Kathleen Lees
First Posted: Dec 08, 2015 03:11 PM EST
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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TagsHealth, Human, Disease, Rare, Rare Disease, Kanuma, patients, Age, Pediatric, Infants, Children, Kids, Adults, Alexion Pharmaceuticals, Lysosomal acid lipase, LAL-D, Spleen, liver, Survival, premature death, Study ©2024 ScienceWorldReport.com All rights reserved. Do not reproduce without permission. The window to the world of science news.
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First Posted: Dec 08, 2015 03:11 PM EST
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.
Related Articles
Lewy Body Dementia 3-Minute Test Helps Diagnose The Rare Disease
Alzheimer's Disease May Be Transmittable
For more great science stories and general news, please visit our sister site, Headlines and Global News (HNGN).
See Now: NASA's Juno Spacecraft's Rendezvous With Jupiter's Mammoth Cyclone