IntraBio Announces Submission of Supplemental New Drug Application for Levacetylleucine for Ataxia-Telangiectasia
AUSTIN, Texas--( BUSINESS WIRE)--IntraBio Inc. today announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for levacetylleucine for the treatment of Ataxia-Telangiectasia (A-T), a rare, progressive, inherited neurodegenerative disorder.
This submission represents the first regulatory application submitted to the U.S. Food and Drug Administration seeking approval of a therapy for the treatment of Ataxia-Telangiectasia.
The sNDA seeks to expand the label of levacetylleucine, marketed as AQNEURSA®, to include A-T. AQNEURSA® is currently approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.
The submission is supported by data from a pivotal Phase III clinical trial evaluating levacetylleucine in adult and pediatric patients with A-T. In this randomized, double-blind, placebo-controlled, crossover study, levacetylleucine met its primary endpoint and key secondary endpoints with high statistical significance. Levacetylleucine was generally safe and well-tolerated, with no drug-related serious adverse events observed, consistent with its established safety profile.
About Ataxia-Telangiectasia
Ataxia-Telangiectasia (A-T) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. It is characterized by degeneration of the cerebellum, resulting in worsening loss of coordination, impaired speech, abnormal eye movements, and eventual wheelchair dependence. Many patients also develop telangiectasia, immune deficiency with recurrent and potentially life-threatening infections, lung disease, and a markedly increased risk of cancer. There are currently no approved therapies for A-T.
About AQNEURSA®
U.S Indication
AQNEURSA® (levacetylleucine) is approved in the United States for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing at least 15 kg.
U.S. IMPORTANT SAFETY INFORMATION
Embryo-Fetal Toxicity
Pregnancy and Lactation
Adverse Reactions
Drug Interactions
To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please click here for Full US Prescribing Information for AQNEURSA: https://www.aqneursahcp.com/wp-content/prescribing-information.pdf
EMA Indication
AQNEURSA® is authorized in the European Union for the treatment of neurological manifestations of Niemann-Pick disease type C in adults and children aged 6 years and older weighing at least 20 kg, either in combination with miglustat or as monotherapy in patients who cannot tolerate miglustat. EMA Indication and Important Safety Information
About IntraBio
IntraBio Inc. is a global biopharmaceutical company headquartered in Austin, Texas, focused on developing and commercializing targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio’s platform technologies are built on decades of scientific research and collaboration with leading universities and institutions worldwide, including the University of Oxford and the University of Munich.