APLT - Applied Therapeutics stock rises on orphan drug status in EU for AT-007 for metabolic disorder
The European Medicines Agency (EMA) granted orphan medicinal product designation to Applied Therapeutics' (NASDAQ:APLT) AT-007 (gavorestat) to treat Galactosemia. Galactosemia is a rare, genetic metabolic disorder which affects the body's ability to convert galactose to glucose. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, causing neurological complications, including issues with speech, cognition, behavior, and motor skills, and also juvenile cataracts and ovarian insufficiency in women. There are about 4K patients with Galactosemia in the EU and 120 new births per year, according to the company. "We plan to meet with the EMA in the third quarter to discuss a potential MAA submission in Europe for conditional approval based on available biomarker data or for full approval based on expected clinical outcomes data," said Applied Founder and CEO Shoshana Shendelman. The EMA's orphan product status provides certain incentives, including 10 years of market exclusivity if the drug is approved and on the
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Applied Therapeutics stock rises on orphan drug status in EU for AT-007 for metabolic disorder