Applied Therapeutics, Inc. has announced that their drug candidate, AT-007 (Govorestat), has received orphan medicinal product designation from the European Medicines Agency (EMA) for the treatment of Sorbitol Dehydrogenase (SORD) Deficiency. This designation reflects the high unmet medical need in SORD Deficiency and recognizes the benefit of Govorestat in reducing toxic sorbitol levels. New data published in the Journal of Clinical Investigation has further explained the molecular pathophysiology of SORD neuropathy and the role of sorbitol in neuronal toxicity. This is an important step for advancing regulatory initiatives in Europe and provides certain benefits and incentives for the development and marketing of the medicine.
Pediatric CMT Natural History Study enhancement to their Charcot-Marie-Tooth (CMT) and Inherited Neuropathies (IN) patient registry, Global Registry for Inherited Neuropathies (GRIN).