FDA Approves IntraBio’s Aqneursa for Niemann-Pick Disease Type C, Marking Second Approval in a Week

Approval:
The FDA approved IntraBio's Aqneursa (levacetylleucine) for treating neurological symptoms associated with Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kilograms.

Second Approval in a Week:
This approval comes shortly after the FDA approved Zevra Therapeutics' Miplyffa (arimoclomol) for the same condition, highlighting the agency's commitment to supporting the development of new treatments for rare diseases.

Clinical Trial:
The approval was based on a randomized, double-blind, placebo-controlled, two-period, 24-week crossover study that demonstrated the efficacy and safety of Aqneursa in improving neurological symptoms in NPC patients.

Rare Disease:
Niemann-Pick disease type C is an ultra-rare genetic disorder that affects approximately one in every 150,000 people, leading to progressive neurological symptoms and organ dysfunction, with patients typically living only up to around 13 years.

Treatment Details:
Aqneursa is taken orally up to three times per day, with or without food, and the recommended dose varies depending on the individual's body weight. Common side effects include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.

Regulatory Designations:
Aqneursa received Priority Review, Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.