NUZ inks positive opinion on orphan product designation for NUZ-001 in Europe

Neurizon has received a notice of positive opinion for orphan medicinal product designation for lead drug NUZ-001
The notice was received from the European Medicines Agency
The move is a critical milestone and highlights the potential of NUZ-001 to provide a meaningful therapeutic option

Special report: Neurizon has been buoyed by the receipt of a positive opinion from the European Medicines Agency in relation to its application for orphan medicinal product designation (OMPD) for NUZ-001 to treat amyotrophic lateral sclerosis (ALS).

Clinical stage bio-tech company Neurizon Therapeutics (ASX:NUZ) is dedicated to advancing treatments for neurodegenerative diseases.

Its lead drug candidate, NUZ-001, has been developed to target ALS, the most common form of motor neurone disease.

Neurizon’s strategy is to accelerate access to effective ALS treatments for patients while exploring NUZ-001’s potential for broader neurodegenerative applications.

Through international collaborations and rigorous clinical programs, Neurizon is dedicated to creating new horizons for patients and families impacted by complex neural disorders.

Notice of positive opinion from EMA

NUZ has received notice of a positive opinion from the European Medicines Agency (EMA) for Orphan Medicinal Product Designation (OMPD) following its application for NUZ-001.

The European Commission is scheduled to issue the official decision on the Orphan Designation in December.

OMPD will offer Neurizon a range of attractive incentives for NUZ-001 including reduced regulatory fees, free protocol assistance, and importantly, 10 years of market exclusivity in the European Union.

During the exclusivity period, the EMA and the EU Member States will not accept another marketing authorisation application for a similar medicinal product in the same therapeutic indication.

‘Market exclusivity’

“Receiving a positive opinion from the EMA for Orphan Medicinal Product Designation is a critical milestone for Neurizon,” NUZ managing director Dr Michael Thurn said.

“The prevalence in the EU is double that of the United States.

“With the OMPD, along with the Orphan Drug Designation from the United States Food and Drug Administration, we have secured market exclusivity for NUZ-001 across the world’s key markets for the treatment of ALS.

“This important recognition highlights the significant potential of NUZ-001 to provide a meaningful therapeutic option for patients with ALS while building the commercial value of this promising candidate.”

What’s next?

NUZ is advancing NUZ-001 through a Phase 2/3 clinical study as part of the HEALEY ALS Platform Trial, with patient enrolment expected to commence in early H1 CY2025.

The company’s protocol regimen has been designed to support the potential for early regulatory approval and the notice of a positive opinion from the EMA on the OMPD further supports its pathway for NUZ-001, underscoring its potential to meet urgent unmet needs in ALS on a global scale.

Next steps include a formal decision from the European Commission in December and continuous regulatory engagement with the EMA and the United States Food and Drug Administration to support the clinical development pathway of NUZ-001.

This article was developed in collaboration with Neurizon Therapeutics, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.