Exciting Advances in Azafaros Clinical Programs for NPC and GM Gangliosidoses
Azafaros has announced significant progress toward global Phase 3 trials for their innovative therapy, nizubaglustat, targeting Niemann-Pick Disease Type C (NPC) and GM gangliosidoses. Highlights include:
Regulatory Approvals: Clinical Trial Applications (CTAs) have been approved across multiple European countries. Trials are set to begin in Q2 2025 and will span regions including the United States, Europe, and Latin America.
Orphan Drug Designations: Nizubaglustat has been granted Orphan Drug and Orphan Medicinal Product designations in the US and EU, recognising its potential as a life-changing therapy for these rare diseases.
Innovative Therapy: Nizubaglustat, a small molecule with a unique dual mode of action, is designed to address critical neurological symptoms associated with lysosomal storage disorders like NPC.
This exciting update marks a step closer to new treatment options for families affected by these devastating conditions.
Download the full announcement below. Together, we’re advancing hope for rare diseases.
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