IntraBio's AQNEURSA Approved by U.S. FDA for Niemann-Pick Disease Type C Treatment

IntraBio Inc., a prominent entity in the discovery and development of innovative treatments for rare neurodegenerative diseases, announced that the U.S. Food and Drug Administration (FDA) has given the green light to AQNEURSA™ (levacetylleucine). This approval is specifically for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in both adults and pediatric patients who weigh at least 15 kilograms. AQNEURSA stands out as the sole FDA-approved monotherapy designed to address NPC.

Mallory Factor, the President and CEO of IntraBio, expressed the company’s commitment to developing novel treatments for patients with critical unmet medical needs like NPC. Factor highlighted that the approval of AQNEURSA marks a significant milestone, providing hope to patients and families affected by this devastating condition. He emphasized IntraBio’s dedication to ensuring that all eligible patients can access this new treatment. Furthermore, Factor mentioned the potential of AQNEURSA in treating other rare and common neurodegenerative and neurodevelopmental disorders, with ongoing efforts to develop the drug for additional indications.

NPC is a rare, inherited lysosomal storage disorder affecting approximately 1 in 100,000 live births. Individuals with NPC often endure a range of systemic, neurological, and psychiatric symptoms, severely impacting their functional abilities. Prior to this approval, existing treatments failed to address these debilitating symptoms effectively.

Laurie Turner, Family Services Manager at the National Niemann-Pick Disease Foundation, hailed the FDA's approval of AQNEURSA as a pivotal breakthrough for the NPC community. She acknowledged the long-awaited arrival of an effective treatment and expressed gratitude for the innovative research efforts that led to this development. Turner also conveyed readiness to support families as they begin this new treatment journey.

The FDA’s approval of AQNEURSA is based on data from the IB1001-301 clinical trial. This multinational, randomized, double-blind, placebo-controlled study assessed the drug’s impact on neurological symptoms in pediatric and adult patients diagnosed with NPC. The trial, which involved 60 participants, successfully met both its primary and secondary efficacy endpoints.

Results from the trial indicated significant improvements in neurological signs and symptoms, with functional benefits becoming apparent within 12 weeks. These findings were published in the February 1, 2024, edition of the New England Journal of Medicine. The study focused on a modified version of the Scale for the Assessment and Rating of Ataxia (SARA), known as the functional SARA (fSARA), which evaluates gait, stability, speech, and limb coordination. Patients treated with AQNEURSA showed a mean treatment difference of -0.4 in their fSARA scores compared to those given a placebo, indicating substantial improvements.

AQNEURSA was generally well tolerated during the trial. The most common adverse reactions included abdominal pain, difficulty swallowing, upper respiratory tract infections, and vomiting.

AQNEURSA is indicated for treating the neurological symptoms of NPC in adults and children weighing 15 kilograms or more. However, it carries a risk of embryo-fetal toxicity, based on animal studies, and may harm an unborn child if administered during pregnancy. Women of reproductive potential should confirm they are not pregnant before starting treatment and use effective contraception during and for seven days after completing the treatment.

IntraBio’s patient support program, AQNEURSA Cares™, offers financial and educational resources to help patients access the treatment. The program provides financial assistance to minimize out-of-pocket costs and connects patients with third-party resources.

IntraBio Inc. is a US-based biopharmaceutical company dedicated to creating novel drugs for treating both rare and common neurological diseases. The company’s platform technologies stem from extensive research and collaboration with leading global universities and institutions.