Ann: PharmAust Progresses to Elevated Doses in MND Patients, page-32

From a previous announcement
PharmAust expects that in due course MPL will receive orphan drug designation by the FDA
for the indication of motor neurone disease.
Such designations come with a number of financial and supportive benefits. The Orphan Drug Act provides for granting special status to a drug or
biological product to treat a rare disease or condition upon request of a sponsor.


Acceptance is conditional upon a minor update to the Ethics committee providing the location of laboratories where
biospecimens collected during the trial will be forwarded for analysis.
The consortium is finalising negotiations with several laboratories possessing the required
capabilities. Trial commencement is anticipated for CYQ4 2021.
FightMND has committed more than $48m to research to help find a cure for motor neurone
disease since former AFL player and coach Neale Daniher founded the charity in 2014.

As announced on 21 September 2020, FightMND awarded PharmAust and the Principal
Investigators Dr Susan Mathers of Calvary HealthCare Bethlehem and Professor Dominic
Rowe of Macquarie University $881,085 to undertake this trial. PharmAust and Dr Mathers and
Professor Rowe are grateful for the support from FightMND and look forward to providing trial
outcomes as they become known.

The Great part about applying for Orphan Status is it is a single form
Free to apply
The FDA takes 90 days to process the application
and

A loophole in the Orphan Drug Act allows mainstream medications to be granted orphan drug status when it's discovered that they may be effective against new illnesses. Because orphan drugs are not produced on a vast scale, their scarcity can lead to allocation and pricing disparities. While repurposing drugs can be very advantageous, there needs to be safeguards to protect from price gouging and preventing access to life-saving treatments.

The Benefits of being granted FDA Orphan Status are significant,,

Orphan Drug Designation Awardees are Granted the Following Benefits from the FDA:

• Tax credits of 50% off the clinical drug testing cost awarded upon approval
• Eligibility for market exclusivity for 7 years post approval
• Waiver of new drug application (NDA)/ biologics license application (BLA) application fee (approximately $2.2 million value)

Plus Patients will soon be able to be granted refunds on medications that have been granted under Orphan Drug Status..

This could be the most expedient way to commercialise Pharmapantel...especially as PAA have manufactured to GMP grade Tablets, MPL is a repurposed drug and PAA will have PKD data and a strong indication that MPL will benefit MND sufferers....