PharmAust seeks orphan drug designation in Europe for monepantel to treat ALS

PharmAust submits a request for orphan medicinal product designation to the EMA for monepantel to treat amyotrophic lateral sclerosis
The designation will provide PharmAust with significant regulatory support and commercial benefits
PharmAust received an Orphan Drug Designation from the US FDA for monepantel to treat amyotrophic lateral sclerosis

Special Report: Clinical-stage biotech PharmAust has submitted a request for orphan medicinal product designation (OMPD) to the European Medicines Agency (EMA) for its lead drug monepantel for the treatment of amyotrophic lateral sclerosis (ALS).

PharmAust (ASX:PAA) has completed a pre-submission meeting with the EMA and the Committee for Orphan Medicinal Products (COMP) and has now submitted a request for OMPD for monepantel (MPL) for the treatment of ALS.

PAA said the request for OMPD was made following a pre-submission meeting held in August 2024 with the EMA and COMP.

Encouraged by the EMA, the pre-submission meeting offers companies a valuable opportunity to receive feedback on their draft applications, increasing the likelihood of success following a formal OMPD submission.

PAA has already received an Orphan Drug Designation (ODD) from the US Food and Drug Administration’s (FDA) Office of Orphan Products Development earlier this year for the use of MPL in treating ALS.

The OMPD evaluation period has a fixed duration of 90 days with a decision expected in December 2024.

Commercial and regulatory incentives

Similar to the FDA’s ODD, the OMPD is an EMA initiative to encourage companies to develop safe and effective treatments for rare diseases or neglected severe disorders.

OMPDs provide structured regulatory and commercial incentives to companies, including:

Protocol assistance – Companies can seek advice on the studies needed to demonstrate the medicine’s quality, benefits, and risks, and information on the significant benefit of the medicine.
Central marketing authorisation in the European Union –Companies can make a single application to the EMA, resulting in a single opinion and decision from the European Commission, valid in all EU member states.
Access to conditional approval – There is potential to receive conditional approval under the centralised procedure.
10 years market exclusivity – Approved orphan medicines benefit from ten years of protection from competition with similar medicines for the same indications once approved.
Reduced fees for regulatory activities – This includes reduced fees for protocol assistance, marketing authorisation applications, inspections before authorisation, and changes to marketing authorisations made after approval, as well as reduced annual fees.
SME benefits – Companies with SME status receive further incentives, including administrative and procedural assistance from the Agency’s SME office and fee reductions.

EMA important market for PAA

Managing director Dr Michael Thurn said PAA was encouraged by the feedback received from the EMA on its draft application and the invitation to submit a full OMPD application.

“The evaluation period has a fixed duration of 90 days, during which the COMP will consider our application, assessing clinical data alongside other evidence to determine MPL’s eligibility for OMPD,

“The EU market represents an important market for PharmAust, where the incidence of ALS in European countries is among the highest globally, ranging from 1.5 to 3.8 per 100,000 people.

“In July, we received Small Medium Enterprise (SME) status from the EMA and look forward to advising the market of the outcome of the OMPD by year-end.”

PAA recently released promising interim results from its ongoing open-label extension study of its MPL to treat ALS.

The study showed that MPL administered at a daily dose of 10mg/kg, continues to be well tolerated and slows disease progression while improving survival rates.

Compared to untreated ALS patients from a historical database, those on MPL experienced significantly longer survival, an 80.3% reduced risk of death, and a 43.2% slower rate of functional decline.

This article was developed in collaboration with PharmAust, 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.