European Medicines Agency Committee Recommends Designation for Rare Neurological Condition with No Approved Therapies

IntraBio Inc. today announced that a positive opinion recommending Orphan Medicinal Product Designation for Acetylleucine for the treatment of CACNA1A disorders has been granted by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA). The European Commission is expected to grant the designation in Q2 2026.

CACNA1A-related disorders are rare, genetic neurological conditions characterized by progressive impairment in coordination, balance, speech, and overall neurological function. Currently, no approved therapies exist for these patients.

Acetylleucine is a modified amino acid under development for a range of neurodegenerative and neurodevelopmental disorders. Building on its prior Phase III pivotal trials with levacetylleucine for Niemann-Pick disease type C (NPC) and for Ataxia-Telangiectasia, IntraBio is preparing to initiate a multinational Phase III trial evaluating levacetylleucine for CACNA1A-related disorders in 2026 (ClinicalTrials.gov: NCT07221292).

“We are moving with urgency to initiate our global Phase III clinical trial and to advance the investigation of levacetylleucine for as many rare disease communities as possible,” said Taylor Fields, Chief Development Officer at IntraBio. “We remain committed to rigorous label expansion in rare and complex neurological diseases with huge unmet medical need.”

Levacetylleucine is an investigational product for the treatment of CACNA1A-related disorders. Its safety and efficacy for this specific indication have not been established.

About Orphan Medicinal Product Designation in the European Union

Orphan Medicinal Product Designation in the European Union is granted to therapies intended for the treatment of rare, serious, or life-threatening conditions and provides regulatory incentives, including ten years of market exclusivity following approval, which may be extended to twelve years.

About AQNEURSA®

Levacetylleucine (AQNEURSA®) 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 ≥15 kg.
US Indication and Important Safety Information

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 an Austin, Texas-based global biopharmaceutical company that develops and commercializes targeted therapies for rare and common neurological, neurodevelopmental, and mitochondrial diseases. IntraBio's platform technologies result from decades of research and collaboration with universities and institutions worldwide, and leverage the expertise of its scientific founders from the University of Oxford and the University of Munich.

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