The European Commission has granted Orphan drug status for Alterity Therapeutics’ (ASX:ATH) lead molecule, PBT434, for the treatment of Multiple System Atrophy (MSA).

That gives Alterity 10 years of market exclusivity in the European Union for PBT434 in the treatment of MSA, as well as other benefits including assistance in developing clinical protocols, reduced fees and access to EU-funded research grants.

MSA is a rare neurodegenerative disorder caused by the degeneration of nerve cells in the brain and characterised by tremors, slow movement, muscle rigidity, and postural instability (collectively known as parkinsonism) and ataxia.

Orphan drug status is given to treatments for rare diseases, so called because due to their limited market few pharmaceutical companies pursue research into such products.

“This is an important milestone for Alterity as we advance PBT434 toward patient studies and it follows Orphan Drug designation from the US FDA for treating MSA,” Dr David Stamler, the company’s chief medical officer and senior VP for clinical development, said.

“It reinforces the dire need for treatment options for this particularly debilitating disease and supports our efforts in preparing for our phase two clinical trial.”

Last year the company announced phase one clinical trial results where PBT434 was found to be safe and well tolerated in adult and older adult patients.

It has also been found to inhibit the build up of alpha-synuclein protein in the brain, preserve neurons and improve motor function in pre-clinical models of Parkinson’s and MSA.

 

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