Arcturus Therapeutics’ ARCT-032 gets EU’s orphan medicinal product designation for cystic fibrosis

Arcturus Therapeutics Holdings Inc. (Nasdaq: ARCT), a leading global clinical-stage messenger RNA medicines company, has achieved a significant milestone in the development of treatments for infectious diseases and rare liver and respiratory conditions. The European Commission (EC), influenced by a favorable review from the European Medicines Agency (EMA), has recently granted orphan medicinal product designation to ARCT-032, Arcturus’s innovative product candidate aimed at treating Cystic Fibrosis (CF). This follows a similar recognition by the U.S. Food and Drug Administration (FDA) in November 2023, which also designated ARCT-032 as an orphan drug for the treatment of CF.

Orphan medicinal product designation in the European Union (EU) is awarded to promising therapies intended to prevent or treat rare, life-threatening, or severely debilitating diseases affecting not more than five individuals per 10,000 within the EU. This designation is pivotal for the development of ARCT-032, as it offers Arcturus several advantages, including protocol assistance, fee reductions, access to centralized marketing authorization procedures, research funding opportunities, and a decade of market exclusivity post-approval.

Joseph Payne, President & CEO of Arcturus Therapeutics, expressed his enthusiasm for the designation: “We are pleased to receive orphan medicinal product designation from the EC for ARCT-032, as it represents a significant milestone for our CF program. We will continue to advance ARCT-032 as a potential new treatment option for people with CF regardless of their genetic mutations.”

The acknowledgment comes after the successful completion of the single-ascending-dose segment of the study in healthy adults and the enrollment and completion of the first CF patient in the Phase 1b study part of ARCT-032. Arcturus is poised to release interim Phase 1b data in the first half of 2024, maintaining its commitment to addressing the unmet medical needs of CF patients worldwide.

Cystic fibrosis is a genetic disorder characterized by mutations in the CFTR gene, leading to reduced or absent CFTR protein functionality. This deficiency hampers chloride transport in the airways, resulting in thick mucus that can cause severe respiratory complications. While current CFTR modulator therapies aim to alleviate symptoms by enhancing CFTR channel function, they may not be effective for all CF patients due to genetic variation. ARCT-032, leveraging Arcturus’s LUNAR lipid-mediated aerosolized delivery platform, promises to express a functional CFTR mRNA copy in the lungs, potentially restoring CFTR activity and improving lung health in CF sufferers. This therapeutic approach is backed by comprehensive preclinical data, including studies in rodents, ferrets, primates, and human bronchial epithelial cells, demonstrating its capability to reinstate CFTR expression and function.

The orphan medicinal product designation for ARCT-032 by the European Commission is a testament to the innovative approach Arcturus Therapeutics is taking to combat cystic fibrosis. This designation not only highlights the potential of ARCT-032 as a groundbreaking treatment option but also underscores the importance of continued investment in mRNA technologies for rare diseases. With ARCT-032, Arcturus aims to fill a significant gap in CF treatment, offering hope to patients who may not benefit from existing therapies. The progress of ARCT-032 through clinical trials will be closely watched, as it represents a promising advancement in the fight against this debilitating disease.