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Adrenoleukodystrophy (X-ALD) is a rare and chronically threatening neurodegenerative disease and currently there is no a satisfactory treatment. (Photo: Minoryx).
 03.01.2017

Minoryx Therapeutics receives European Orphan Drug Designation for its lead candidate MIN-102

Minoryx Therapeutics – a drug development company specialized in the discovery and development of new drugs for orphan diseases, which has R& D laboratories at the Barcelona Science Park–  today announces that its lead compound MIN-102 has been granted Orphan Drug Designation by the European Medicines Agency (EMA). This designation is granted to medicines that are aimed at a condition that is life-threatening or chronically debilitating and have a prevalence rate in the EU of no more than five in 10,000 and brings a number of benefits to Minoryx including ten years market exclusivity.

 

MIN-102 is a selective PPAR gamma agonist with a superior profile for central nervous system related diseases. It has shown robust preclinical proof of concept in multiple animal models. Phase I studies were initiated based on these results. A phase 2/3 trial in adult AMN patients will be launched during the first half of 2017.

Minoryx Therapeutics’ MIN-102 targets X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease. Currently, there are no pharmacological treatments for X-ALD. MIN-102 is the only product in development for potential use across all the main phenotypes.

“We are pleased to have received European Orphan Drug Designation for MIN-102. This represents an important milestone,” said Marc Martinell, CEO of Minoryx. “We are committed to progressing this drug candidate rapidly with the aim of providing a pharmacological treatment option for caregivers and for X-ALD patients.”

European Orphan Drug Designation is granted to medicines that are aimed at a condition that is life-threatening or chronically debilitating and have a prevalence rate in the European Union of no more than five in 10,000.

Orphan Drug Designation by the European Medicines Agency brings a number of benefits to drug developers, including protocol assistance, prioritized scientific advice, ten years of market exclusivity after marketing the drug and potential regulatory fee reductions.