Neurizon Therapeutics Gains Orphan Drug Status for NUZ-001 in Europe

Neurizon Therapeutics Limited, a clinical-stage biotech firm based in Melbourne, Australia, has received a positive opinion from the European Medicines Agency (EMA) for Orphan Medicinal Product Designation (OMPD) for its lead drug candidate, NUZ-001. This drug is being developed for the treatment of Amyotrophic Lateral Sclerosis (ALS), a severe neurodegenerative disease with limited treatment options.

The EMA's OMPD provides several significant incentives for Neurizon. Among these benefits are reduced regulatory fees, free protocol assistance, and a decade of market exclusivity in the European Union (EU). During this exclusivity period, no other marketing authorisation applications for similar medicinal products in the same therapeutic indication will be accepted by the EMA and the EU Member States. The official decision on this OMPD is expected to be issued by the European Commission in December 2024.

Neurizon is progressing NUZ-001 through a Phase 2/3 clinical study as part of the HEALEY ALS Platform Trial, with patient enrolment anticipated to begin in the first half of 2025. The study's protocol regimen is structured to support the potential for early regulatory approval. The positive opinion from the EMA on the OMPD reinforces the pathway for NUZ-001, emphasizing its potential to meet urgent unmet needs in ALS globally.

Dr. Michael Thurn, Managing Director and Chief Executive Officer of Neurizon, remarked that receiving this positive opinion from the EMA is a critical milestone for the company. He noted that the prevalence of ALS in the EU is double that of the United States. With the OMPD, alongside the Orphan Drug Designation from the U.S. Food and Drug Administration, Neurizon has secured market exclusivity for NUZ-001 in the key global markets for ALS treatment. This recognition underscores the significant potential of NUZ-001 to offer a meaningful therapeutic option for ALS patients while enhancing the commercial value of this promising candidate. Dr. Thurn expressed anticipation for further engagement with the EMA and other regulators as the company advances its mission to provide innovative treatments for patients battling this devastating disease.

In the coming months, Neurizon will await the formal decision from the European Commission, expected in December 2024. The company will continue its regulatory engagement with the EMA and the U.S. Food and Drug Administration to support the clinical development pathway of NUZ-001. Additionally, Neurizon will finalize preparations for the Phase 2/3 clinical study under the HEALEY ALS Platform Trial, in line with its strategic focus on neurodegenerative diseases.

Neurizon Therapeutics Limited is committed to advancing treatments for neurodegenerative diseases, with a primary focus on ALS, the most common form of motor neurone disease. The company's strategy aims to accelerate access to effective ALS treatments for patients while also exploring NUZ-001's potential for broader applications in neurodegenerative conditions. Through international collaborations and rigorous clinical programs, Neurizon aims to create new opportunities for patients and families affected by complex neural disorders.