{"id":17053,"date":"2026-06-05T20:09:00","date_gmt":"2026-06-05T18:09:00","guid":{"rendered":"http:\/\/stocks-future.com\/?guid=96f7ce1fb8f4b4f025caad3ed1dc945b"},"modified":"2026-06-05T20:09:00","modified_gmt":"2026-06-05T18:09:00","slug":"intrabio-announces-submission-of-variation-application-to-the-european-medicines-agency-for-aqneursa-for-ataxia-telangiectasia","status":"publish","type":"post","link":"https:\/\/stocks-future.com\/?p=17053","title":{"rendered":"IntraBio Announces Submission of Variation Application to the European Medicines Agency for AQNEURSA\u00ae for Ataxia-Telangiectasia"},"content":{"rendered":"

\nIf approved, <\/i>AQNEURSA\u00ae would become the first approved therapy for Ataxia-Telangiectasia in the European Economic Area<\/i><\/p>

AUSTIN, Texas--(BUSINESS WIRE)--IntraBio Inc. today announced the submission of a variation application to the European Medicines Agency (EMA) to expand the approved Marketing Authorization Application (MAA) for AQNEURSA\u00ae<\/span> (levacetylleucine) to include the treatment of Ataxia-Telangiectasia (A-T).<\/p><\/a><\/a>

\nThis submission represents the first regulatory application submitted to the EMA seeking approval of a therapy for the treatment of A-T, a rare, progressive, inherited neurodegenerative disorder. The variation application is subject to EMA validation before the start of formal scientific review.<\/p>

\nIn the United States, IntraBio\u2019s supplemental New Drug Application for AQNEURSA for A-T has been accepted for review by the U.S. Food and Drug Administration and granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of September 19, 2026.<\/p>

\nThe variation application seeks to expand the label of AQNEURSA\u00ae<\/span> to include A-T. AQNEURSA\u00ae<\/span> is currently approved in the European Economic Area 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, and 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.<\/p>

\nAbout Ataxia-Telangiectasia<\/b><\/p>

\nAtaxia-Telangiectasia (A-T) is a rare, inherited, progressive neurodegenerative disorder that typically begins in early childhood. A-T is estimated to affect 1 in 40,000-100,000 people. 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.<\/p>

\nAbout AQNEURSA\u00ae<\/b><\/p>

\nEMA Indication\n<\/i>AQNEURSA\u00ae 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<\/a><\/p>

\nU.S Indication\n<\/i>AQNEURSA\u00ae (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.<\/p>

\nU.S. IMPORTANT SAFETY INFORMATION<\/b><\/p>

\nEmbryo-Fetal Toxicity<\/b><\/p>