ReceptoPharm, has received approval from the UK's Medicines Health and Regulatory Agency (MHRA) for its application of human clinical trials for the treatment of adrenomyeloneuropathy (AMN).
AMN is a rare inherited metabolic disorder characterized by the loss of the fatty covering (myelin sheath) on nerve fibers within the brain (cerebral demyelination) and the progressive degeneration of the adrenal gland (adrenal atrophy). Neurological disability in AMN is slowly progressive over several decades. AMN interests the wider neurologic community because of its similarities to multiple sclerosis (MS). There is currently no approved treatment for AMN. Additionally, the disease's rarity designates it as an orphan drug candidate both in Europe and the U.S. ReceptoPharm's drug, RPI-78M, was assessed in a placebo-controlled, double-blind study in the UK in 2003. The trial, published in the journal Neurology, represented the first controlled study of RPI-78M. The therapy was proven to be safe and well-tolerated.