Amylyx Pharmaceuticals’ AMX0035 Gains the EC’s Orphan Drug Designation to Treat Wolfram Syndrome
Shots:
- Following COMP’s positive opinion, the EC has granted ODD to AMX0035 (oral combination of sodium phenylbutyrate & taurursodiol) to treat Wolfram syndrome
- AMX0035 is being assessed under a P-II (HELIOS) study for its safety, tolerability & efficacy plus for evaluating endocrinological, neurological & ophthalmologic function in adults (n=12) with Wolfram syndrome
- Interim analysis of the study in 8 subjects at wk.24 showed improved pancreatic function & glycemic control; all 8 reached responder criteria, showing either improved or stabilized disease as per PGIC & CGIC scales, with most reporting improved vision. Topline data from all 12 subjects at wk.24 are expected in H2'24
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Disha is a content writer at PharmaShots. She is passionate and curious about recent updates and developments in MedTech and Pharma industry. She covers news related to clinical trial results and updates. She can be contacted at connect@pharmashots.com.
