The US Food and Drug Administration (FDA) has granted orphan drug designation to Minoryx Therapeutics’ lead compound MIN-102.

MIN-102 is a selective PPAR gamma agonist that targets X-linked adrenoleukodystrophy (X-ALD), a rare, chronically debilitating and life-threatening neurodegenerative disease.

The disease primarily affects males and is caused by mutations in the ABCD1 gene. Heterozygous women also develop it later in life.

The two main clinical phenotypes of X-ALD are adrenomyeloneuropathy (AMN), characterised by progressive motor dysfunction, and inflammatory cerebral ALD (cALD), which is characterised by severe neuroinflammation leading to early death.

Minoryx Therapeutics CEO Marc Martinell said: “We are delighted that our lead candidate, MIN-102, now has Orphan Drug Designation from both the FDA and the EMA.

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“These acknowledgements prove that our drug candidate addresses an unmet need in orphan diseases. We are committed to progressing it rapidly through the next phases of drug development in order to offer a pharmacological treatment for X-ALD.”

Minoryx is planning to launch a Phase II/III trial in adult AMN patients during the first half of this year.

“We are delighted that our lead candidate, MIN-102, now has orphan drug designation from both the FDA and the EMA.”

A drug will receive the FDA orphan drug status if it aims at a rare disease or condition that affects less than 200,000 people in the US.

The designation grants seven years of market exclusivity in the country along with other benefits such as tax credits, protocol assistance and research grants.

MIN-102 received orphan drug designation from the European Medicines Agency (EMA) at the end of last year.

The drug is a metabolite of pioglitazone and shows a better brain penetration and safety profile.

Image: Minoryx Therapeutics CEO Marc Martinell. Photo: courtesy of Andrew Lloyd & Associates / Minoryx Therapeutics.