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Minoryx Therapeutics, a Catalan biotechnology firm specializing in the discovery and development of new drugs for minority diseases, has received orphan drug designation from the European Medicines Agency (EMA) for its drug candidate MIN-102, a compound to treat X-linked adrenoleukodystrophy (X-ALD), which is currently untreatable.

Orphan drug designation, which is granted to drugs targeting diseases that affect no more than five in 10,000 individuals in the European Union, gives the company market exclusivity for 10 years.

After having begun phase I clinical trials this year, Minoryx hopes to start phase II/III clinical trials by mid-2017 in adult patients. According to the studies conducted so far, MIN-102 prevents dysfunctions associated with X-ALD, including oxidative stress, neural inflammation, demyelination and axonal degeneration.

This disease, which affects mainly men, is tied to the X chromosome and caused by mutations on the ABCD1 gene. It is characterized by the accumulation of very long chain fatty acids that lead to a highly debilitating and potentially deadly chronic neurodegenerative disorder. The main tissues affected are those in the central nervous system and the adrenal cortex, due to loss of myelin. The effects can lead to two main phenotypes and the Minoryx candidate shows potential in treating both.


For more information, see the press release


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