FDA Approves New Niemann-Pick Type C Drug

Today, the U.S. Food and Drug Administration (FDA) announced the approval of Aqneursa (levacetylleucine) for treating neurological symptoms in Niemann-Pick disease type C (NPC) patients. This includes both adults and children who weigh at least 15 kilograms. Janet Maynard, M.D., M.H.S., the director of the Office of Rare Diseases, Pediatrics, Urologic and Reproductive Medicine at the FDA’s Center for Drug Evaluation and Research, stated that this is the second NPC treatment approved within a week. She emphasized that this approval highlights the FDA’s dedication to advancing treatments for rare diseases, showcasing their collaborative efforts with the scientific community to tackle unique challenges in rare disease drug development.

NPC is a rare genetic disorder that manifests as progressive neurological deterioration and organ dysfunction. The disease stems from mutations in either the NPC1 or NPC2 gene, which are crucial for the intracellular transport of cholesterol and other lipids. When these genes malfunction, cells become dysfunctional, leading to organ damage. The prognosis for NPC patients is grim, with an average life expectancy of just 13 years.

The approval of Aqneursa was based on a comprehensive study evaluating its safety and efficacy. This study was a randomized, double-blind, placebo-controlled, two-period, 24-week crossover trial, with each treatment period lasting 12 weeks. A total of 60 patients participated, all of whom were at least 4 years old with confirmed NPC diagnoses and exhibited at least mild neurological symptoms. Participants were allowed to use miglustat, an enzyme inhibitor, as part of their background treatment during the study.

The primary metric for efficacy was a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), known as the functional SARA (fSARA). The fSARA includes modifications to the gait, sitting, stance, and speech disturbance components of the original SARA, enhancing the scoring responses. On average, patients treated with Aqneursa for 12 weeks demonstrated better fSARA scores compared to those who received a placebo.

The prescribing information for Aqneursa includes a warning about potential embryo-fetal harm if the drug is used during pregnancy. Females are advised to inform their healthcare provider about known or suspected pregnancies before starting Aqneursa. Common side effects associated with the drug include abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.

Aqneursa is administered orally up to three times daily, with or without food. The dosage varies based on the patient’s body weight, as detailed in the prescribing information. The FDA granted Aqneursa several designations, including Priority Review, Fast Track, Orphan Drug, and Rare Pediatric Disease, emphasizing the importance and urgency of this treatment.

The approval of Aqneursa was granted to IntraBio Inc.