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Amylyx Pharmaceuticals Receives Orphan Drug Designation From the European Commission for AMX0035 for the Treatment of Wolfram Syndrome
- New designation follows the U.S. Food and Drug Administration (FDA) Orphan Drug Designation for AMX0035 in Wolfram syndrome granted in 2020 - Topline data
About this update from Amylyx Pharmaceuticals, Inc.
[{"type":"text","content":"\n- New designation follows the U.S. Food and Drug Administration (FDA) Orphan Drug Designation for AMX0035 in Wolfram syndrome granted in 2020\n\n\n- Topline data for all 12 participants from Phase 2 HELIOS trial studying impact of AMX0035 on endocrine, metabolic, and neurodegenerative aspects of Wolfram syndrome anticipated fall 2024\n\n\n CAMBRIDGE, Mass.--(BUSINESS WIRE)--\nAmylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced the European Commission (EC), based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted Orphan Drug Designation for AMX0035, Amylyx’ proprietary, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.) for the treatment of Wolfram syndrome.\n\n\nWolfram syndrome is a prototypical disease of endoplasmic reticulum (ER) stress that is rare, progressive, and monogenic and is characterized by childhood-onset diabetes mellitus, optic nerve atrophy, deafness, diabetes insipidus, and neurodegeneration. There are currently no drugs approved for Wolfram syndrome, and many people with the disease die prematurely with severe neurological disabilities.\n\n\nThe FDA previously granted AMX0035 Orphan Drug Designation for the treatment of Wolfram syndrome in 2020. The EMA grants Orphan Drug Designation status for products intended for the treatment, prevention, or diagnosis of rare, life-threatening, or chronically debilitating conditions where the product may represent a significant benefit over existing treatments.\n\n\nAmylyx recently presented positive data from an interim analysis of its Phase 2 HELIOS study, including eight participants with Wolfram syndrome assessed at Week 24, which demonstrated that AMX0035 improved pancreatic function and glycemic control, as measured by C-peptide, HbA1c, and other markers of glucose metabolism. All eight participants met prespecified responder criteria showing either improvement or stabilization of disease according to both the Patient Reported Global Impression of Change (PGIC) and the Clinical Reported Global Impression of Change (CGIC) scales. The majority of participants reported some improvement in vision. In Wolfram syndrome, progressive decline and worsening of outcomes would h...