Levacetylleucine

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 Based on findings from animal reproduction studies, AQNEURSA may cause embryo-fetal harm when administered during pregnancy. The decision to continue or discontinue AQNEURSA treatment during pregnancy should consider the female’s need for AQNEURSA, the potential drug-related risks to the fetus, and the potential adverse outcomes from untreated maternal disease. Pregnancy and Lactation For females of reproductive potential, verify that the patient is not pregnant prior to initiating treatment with AQNEURSA. Advise females of reproductive potential to use effective contraception during treatment with AQNEURSA and for 7 days after the last dose if AQNEURSA is discontinued. There are no data on the presence of levacetylleucine or its metabolites in either human or animal milk, the effects on the breastfed infant or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for AQNEURSA and any potential adverse effects on the breastfed infant from levacetylleucine or from the underlying maternal condition. Adverse Reactions The most common adverse reactions (incidence ≥5% and greater than placebo) are abdominal pain, dysphagia, upper respiratory tract infections, and vomiting. Drug Interactions Avoid concomitant use of AQNEURSA with N-acetyl-DL-leucine or N-acetyl-D-leucine. The D-enantiomer, N-acetyl-D-leucine, competes with levacetylleucine for monocarboxylate transporter uptake, which may reduce the levacetylleucine efficacy. Monitor more frequently for P-gp substrate related adverse reactions when used concomitantly with AQNEURSA; AQNEURSA inhibits P-gp; however, the clinical significance of this finding has not been fully characterized. To report SUSPECTED ADVERSE REACTIONS, contact IntraBio Inc. at 1-833-306-9677 or FDA at 1-800-FDA-1088 or . Please click here for Full US Prescribing Information for AQNEURSA: 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. Contacts For further information, please contact: Cass Fields Vice-President of External Affairs ccfields@intrabio.com