PharmAust seeks European orphan drug designation for MND/ALS drug monepantel
PharmAust (ASX: PAA) has requested the European Medicines Agency (EMA) grant orphan medicinal product designation (OMPD) for monepantel (MPL), its lead candidate in the treatment of amyotrophic lateral sclerosis (ALS) and motor neurone disease (MND).
The OMPD is an EMA initiative to encourage companies to develop safe and effective treatments for rare diseases or neglected severe disorders.
A successful designation can provide incentives such as protocol assistance, central marketing authorisation within the European Union (EU), access to conditional approval and 10 years of market protection from competition with similar medicines for similar indications.
Commercial pathway
The request follows a pre-submission meeting in August with the EMA and the Committee for Orphan Medicinal Products, where PharmAust sought feedback on its application to increase the likelihood of success.
PharmAust managing director Dr Michael Thurn said an OMPD would play a critical role in advancing MPL towards commercialisation.
“The EU represents an important market for us, where the incidence of ALS is amongst the highest worldwide and affects up to 3.8 patients per 100,000 people,” he said.
“OMPD status will complement a small and medium enterprise status received from the EMA in July [and] we look forward to advising the market of the outcome.”
PharmAust’s submission will be evaluated over a 90-day period, with a final decision expected before year-end.
FDA designation
PharmAust received an orphan drug designation (ODD) in May from the US Food and Drug Administration for the use of MPL in the treatment of ALS and MND.
The company will benefit from US incentives including tax credits, grants, a waiver of some administrative fees for clinical trials and seven years of market exclusivity following drug approval.
PharmAust’s application was based on preclinical mechanistic data which demonstrated MPL could induce autophagy in diseased cells and considered the pathology of the disease.
The ODD achievement was referred to as an “outstanding milestone” that would provide a strong pathway forward for the drug.