Pharmaust Granted Orphan Drug Designation For Monepantel In USA

PharmAust Ltd (ASX: PAA) shares rose after securing Orphan Drug Designation (ODD) for its drug monepantel (MPL) from the US FDA.

The ODD has been granted for MPL and its treatment of Motor Neurone Disease (MND) / Amyotrophic Lateral Sclerosis (ALS).

Seven years of market exclusivity

The FDA grants ODD status to assist and encourage companies to develop safe and effective treatments for rare diseases and disorders.

Importantly, the ODD status provides incentives, including tax credits, grants and seven years of market exclusivity following drug approval.

PharmAust’s application was based on data that demonstrates MPL can induce autophagy in diseased cells, and consideration of the pathology of the disease.


PharmAust initially submitted the request for ODD to the FDA in November 2023.

In January 2024, the FDA responded, requesting further supporting data to establish the drug’s potential for effectiveness in MND/ALS.

In March, PharmAust submitted the positive clinical data from its Phase 1 MEND study involving 12 patients with MND/ALS.

Meaningful therapeutic effect

The positive top-line data from the Phase 1 MEND Study was based on daily administration of MPL over a 7 to 12-month period.

This was well-tolerated and did not result in any dose-limiting toxicities or serious adverse effects.

Critically, the daily administration of MPL resulted in a clinically meaningful therapeutic effect.

“Outstanding milestone”

 PharmAust Managing Director John Clark said: “This is an outstanding milestone for PharmAust and monepantel, providing an even stronger pathway forward for the drug, particularly in light of recent failures of other MND/ALS treatments.

 “We are now increasingly optimistic as we progress to our pivotal registration adaptive Phase 2/3 study which will commence in H2 CY2024.”



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