Minoryx Therapeutics announced that the European Commission has granted orphan drug designation to MIN-101 for the treatment of X-linked Adrenoleukodystrophy (ALD). MIN-101 is based on Pioglitazone, which showed excellent efficacy in animal models of ALD.
X-linked adrenoleukodystrophy (ALD) is a rare inherited neurodegenerative disorder, which is chronically debilitating and life threatening. The disease is caused by mutations in the ABCD1 gene located on the X-chromosome that encodes the peroxisomal membrane protein ALDP which is involved in the transmembrane transport of very long-chain fatty acids (VLCFA; ≥C22). A defect in ALDP results in elevated levels of VLCFA in plasma and tissues including the white matter of the brain, the spinal cord and adrenal cortex. The clinical spectrum of ALD ranges from isolated adrenocortical insufficiency and slowly progressive myelopathy to devastating cerebral demyelination, leading to paralysis, dementia and death.