Applied Therapeutics Receives Orphan Medicinal Product Designation from the EMA for AT-007 for Treatment of Galactosemia
NEW YORK, June 03, 2022 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT), a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need, today announced designation of AT-007 (gavorestat) as an orphan medicinal product by the European Medicines Agency (EMA) for treatment of Galactosemia (Galactosaemia).
“We are pleased that the EMA has recognized the high unmet medical need in Galactosemia, the role of galactitol as the toxic metabolite responsible for long-term complications in Galactosemia, and the potential benefit of AT-007 treatment in reducing toxic galactitol levels,” said Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics. “Orphan designation for AT-007 marks an important step towards advancing our regulatory initiatives in Europe. 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.”
Orphan medicinal product designation provides certain benefits and incentives in the EU, including protocol assistance, fee reductions, and ten years of market exclusivity once the medicine is on the market.
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). There are approximately 4,000 patients with Galactosemia in the EU and 120 new births per year.
AT-007 is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. In clinical trials, AT-007 significantly reduced plasma galactitol levels vs. placebo in adults and children with Galactosemia. AT-007 is currently being studied in a Phase 3 clinical outcomes trial (ACTION-Galactosemia Kids) in children ages 2-17 with Galactosemia, as well as a long-term open-label study in adults with Galactosemia. In a pilot study, AT-007 significantly reduced blood sorbitol levels in adults with SORD Deficiency. AT-007 is currently being studied in a Phase 3 trial (INSPIRE) investigating biomarker efficacy, clinical outcomes, and significantly reduced blood sorbitol levels in adults with SORD Deficiency. AT-007 has received both Orphan Drug and Pediatric Rare Disease designations from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The Company’s lead drug candidate, AT-007, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG. The Company is also developing AT-001, a novel potent ARI, for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of Diabetic retinopathy, as well as novel dual PI3k inhibitors in preclinical development for orphan oncology indications.
To learn more, please visit www.appliedtherapeutics.com and follow the company on Twitter @Applied_Tx.
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Applied Therapeutics, Inc.