The FDA has approved IntraBio’s Aqneursa (levacetylleucine), for the treatment of neurological symptoms associated with Niemann-Pick disease type C (NPC).
NPC is caused by mutations in the NPC1 or NPC2 genes, disrupting cholesterol and lipid transport within cells and leading to organ damage. Most individuals with the disease have a significantly reduced life expectancy, often surviving only about 13 years. The approval marks the second NPC treatment approved by the FDA in a week.
Aqneursa’s effectiveness was demonstrated in a 24-week, randomized, double-blind, placebo-controlled study involving 60 patients with confirmed NPC diagnoses. The study used a modified version of the Scale for the Assessment and Rating of Ataxia (SARA) to measure neurological function. Patients treated with Aqneursa for 12 weeks showed significant improvements in their functional SARA scores compared to those on placebo.
The drug works by modulating intracellular pathways that are involved in lipid metabolism. Although the precise mechanism of action for treating NPC remains unclear, it is thought to help stabilize neuronal function and improve the transport of cholesterol and other lipids within cells. This transport is disrupted in NPC, leading to lipid accumulation and cell damage. By addressing these imbalances, Aqneursa may help reduce the neurological symptoms associated with the disease, such as motor dysfunction and cognitive decline.
Earlier this week, the FDA approved Zevra Therapeutics’ Miplyffa (arimoclomol) for the treatment of NPC. Miplyffa, used in combination with miglustat, is the first FDA-approved therapy and was cleared after demonstrating modest clinical benefits and a favorable safety profile.
