Minoryx compound MIN-101 receives orphan drug designation
The drug treats Adrenoleukodystrophy, a rare disease that affects one in 20.000 people
Minoryx Therapeutics, a company specializing in the treatment of minority diseases, has been granted orphan drug designation by the European Medicines Agency (EMA) for their compound MIN-101 to treat Adrenoleukodystrophy, a hereditary neurodegenerative disease that has no cure.
Adrenoleukodystrophy affects 1 in 20,000 people in the world, according to MedlinePlus. It affects mainly men and causes very long-chain fatty acids to accumulate in the nervous system, adrenal glands and testicles. This alteration can lead to loss of muscle tone, strabismus, blindness, convulsions and the progressive deterioration of the nervous system.
Designation as an orphan drug facilitates development and cuts costs. And, if it reaches the market, the drug would have market exclusivity for 10 years. Thus, receiving this designation helps compounds that otherwise would have difficulties finding investors to fund them.
Minoryx Therapeutics, located at Parc TecnoCampus Mataró-Maresme, was founded in 2011 and in 2013 won the EmprenedorXXI award, which is promoted by “la Caixa”, the Barcelona City Council and Biocat. In summer of 2013, another company in the BioRegion also received this designation: Oryzon with its compound ORY-1001 to treat leukemia.
More information is available in the Minoryx Therapeutics press release.
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