Neurizon Therapeutics Limited has received a positive opinion from the European Medicines Agency (EMA) for Orphan Medicinal Product Designation (OMPD) for its lead drug candidate, NUZ-001, in the treatment of Amyotrophic Lateral Sclerosis (ALS). The European Commission is expected to issue the official decision on the Orphan Designation in December.
This designation provides Neurizon with several incentives, including reduced regulatory fees, free protocol assistance, and 10 years of market exclusivity in the European Union (EU). During this period, the EMA will not accept marketing authorization applications for similar medicinal products targeting the same therapeutic indication.
Clinical Development of NUZ-001
Neurizon is currently advancing NUZ-001 through a Phase 2/3 clinical study as part of the HEALEY ALS Platform Trial. Patient enrollment is anticipated to commence in early H1 CY2025. The trial protocol is designed to potentially support early regulatory approval. According to Dr. Michael Thurn, Managing Director and CEO, the OMPD, along with the Orphan Drug Designation from the United States Food and Drug Administration, secures market exclusivity for NUZ-001 in key global markets for ALS treatment.
Significance of Orphan Drug Designation
The Orphan Medicinal Product Designation underscores NUZ-001's potential to address the unmet needs in ALS treatment. The prevalence of ALS in the EU is notably higher than in the United States. This recognition highlights the potential of NUZ-001 as a meaningful therapeutic option for patients with ALS, while also enhancing the commercial value of the drug candidate.
About NUZ-001
NUZ-001 is being developed by Neurizon Therapeutics as a treatment for ALS, the most common form of motor neurone disease. Neurizon's strategy focuses on accelerating access to effective ALS treatments and exploring NUZ-001's potential for broader neurodegenerative applications.
