PharmAust (ASX: PAA) is pursuing European regulatory support for its lead drug candidate, monepantel (MPL), in the treatment of amyotrophic lateral sclerosis (ALS), also known as motor neurone disease (MND). The company has submitted a request to the European Medicines Agency (EMA) for Orphan Medicinal Product Designation (OMPD) for MPL.
The OMPD is an EMA initiative designed to encourage the development of safe and effective treatments for rare and severe diseases. If granted, the designation would provide PharmAust with several incentives, including protocol assistance, centralized marketing authorization within the European Union, potential for conditional approval, and 10 years of market protection from competition for similar medicines targeting the same indication.
Strategic Regulatory Pathway
PharmAust's application follows a pre-submission meeting with the EMA and the Committee for Orphan Medicinal Products (COMP) in August 2024. This meeting allowed PharmAust to gain feedback on its application, increasing the likelihood of a successful designation. The EMA's evaluation period for the OMPD is a fixed 90 days, with a decision anticipated before the end of 2024.
According to PharmAust managing director Dr. Michael Thurn, securing OMPD status is crucial for advancing MPL towards commercialization in the EU, where ALS incidence is among the highest globally, affecting up to 3.8 patients per 100,000 people.
Parallels with FDA Orphan Drug Designation
In May, PharmAust received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for MPL in the treatment of ALS and MND. This designation allows PharmAust to benefit from U.S. incentives, including tax credits, grants, waived administrative fees for clinical trials, and seven years of market exclusivity post-approval.
The FDA's decision was supported by preclinical data demonstrating MPL's ability to induce autophagy in diseased cells, addressing the underlying pathology of ALS.
Clinical Evidence and Ongoing Studies
PharmAust recently reported positive interim results from an ongoing open-label extension study of MPL in ALS. The study indicated that MPL, administered at a daily dose of 10mg/kg, was well-tolerated and slowed disease progression while improving survival rates. Compared to untreated ALS patients from a historical database, those on MPL experienced a significantly longer survival, an 80.3% reduced risk of death, and a 43.2% slower rate of functional decline.
