Tag: Amino acid derivative

In September 2024, the U.S. Food and Drug Administration (FDA) approved Aqneursa (levacetylleucine) for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC), classifying it as a modified amino acid. This orally administered medication addresses the progressive neurological decline seen in this rare genetic disorder.

In September 2024, the U.S. Food and Drug Administration (FDA) approved Aqneursa, the brand name for the medication levacetylleucine, as a treatment for the neurological manifestations of Niemann-Pick disease type C (NPC). This marked a significant milestone for the rare disease community by providing a new therapeutic option for a debilitating condition.