FDA Approves Miplyffa for the Treatment of Niemann-Pick Disease Type C
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September 26, 2024September 25, 2024 – IntraBio Inc. announced the U.S. Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing ≥15kg.
- Aqneursa is the only FDA-approved, stand-alone therapy indicated for the treatment of NPC. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives.
- NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological, and psychiatric symptoms that can be debilitating and significantly impact functional abilities.
- Source: IntraBio Inc.