FDA approval for Aqneursa ( levacetylleucine) to treat Niemann-Pick disease Type C
IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases, announced that the FDA has approved Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing greater than 15 kg. Aqneursa is the only FDA-approved stand-alone therapy indicated for the treatment of NPC.
NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives.
The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial (NCT05163288), which evaluated the impact of Aqneursa on neurological symptoms and functioning in pediatric (aged 4 years and older) and adult patients (n=60) with a confirmed diagnosis of NPC.
The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving Aqneursa. Results from the study showed Aqneursa significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks. These findings were published in the February 1, 2024 issue of the New England Journal of Medicine.
Citation:Trial of N-Acetyl-l-Leucine in Niemann–Pick Disease Type C.Authors: Tatiana Bremova-Ertl, M.D., Ph.D. , Uma Ramaswami, M.D. Marion Brands, M.D. Tomas Foltan, M.D., Matthias Gautschi, M.D., Ph.D. , Paul Gissen, M.D. , Francesca Gowing, M.D., +14, and Kyriakos Martakis, M.D..Published January 31, 2024. N Engl J Med 2024;390:421-431DOI: 10.1056/NEJMoa2310151 .VOL. 390 NO. 5
Learning Zones
The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.
1 mins