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Minoryx Therapeutics, which works to discover new drugs for minority diseases, has been granted orphan drug designation by the United States Food and Drug Administration (FDA) for its drug candidate MIN-102, which targets a currently untreatable disease of the central nervous system.

The compound, which was also granted orphan drug designation by the EMA (homologous European agency) in late 2016, hopes to treat X-linked adrenoleukodystrophy (X-ALD) and now has seven years’ market exclusivity in the United States, as well as other perks like tax breaks, help with protocols and research grants. These advantages are on top of the 10 years’ market exclusivity the company already has in Europe.

In the first half of 2017, Minoryx hopes to begin phase II/III clinical trials in adult patients with adrenomyeloneuropathy, one of the main clinical phenotypes of X-ALD.


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