The pharmaceutical company Intrabio, who developed the recently FDA-approved drug AQNEURSA for people with Niemann-Pick Disease Type C, has announced their plans to begin a phase III trial of the drug in SCA6 and EA2.Â
In September 2024, the US drug regulator the FDA approved their symptom-relief drug AQNEURSA (levacetylleucine) for the treatment of Niemann-Pick Disease Type C (NPC). This drug is under review for approval for NPC by the European drug regulator, the EMA. Read more about this here.
Intrabio has announced that they plan to trial this drug in CACNA1A-related disorders, which includes SCA6 and EA2. They hope to be able to start the trial in 2026, and are exploring potential UK sites.
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