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FDA Approves Second Treatment for Niemann-Pick Disease, Type C

Today, FDA approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg. Aqneursa is the second treatment FDA has approved for NPC within the span of a week underscoring the agency’s commitment to support the development of new treatments for rare diseases.

NPC is a rare genetic disease that results in progressive neurological symptoms and organ dysfunction. It is caused by variants in either the NPC1 or NPC2 gene, resulting in impaired intracellular transport of cholesterol and other lipids. The average lifespan for people with NPC is 13 years.

The most common side effects of Aqneursa are abdominal pain, dysphagia (difficulty swallowing), upper respiratory tract infections and vomiting. See full prescribing information for additional information on the safety and effectiveness of Aqneursa.

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