Applied Therapeutics Updates on Govorestat for Classic Galactosemia Treatment

Applied Therapeutics, Inc., a clinical-stage biopharmaceutical company listed on Nasdaq under the ticker APLT, has shared an update on the ongoing review of its New Drug Application (NDA) for govorestat, aimed at treating Classic Galactosemia. Recently, the company completed a late-cycle review meeting with the United States Food and Drug Administration (FDA), where it was communicated that an Advisory Committee meeting, initially scheduled for October 9, 2024, would no longer be necessary. The FDA confirmed that the Priority Review of the NDA is progressing as planned, with expectations to finalize post-marketing requirements by October 2024. The Prescription Drug User Fee Act (PDUFA) target action date remains set for November 28, 2024.

Dr. Shoshana Shendelman, the Founder and CEO of Applied Therapeutics, expressed satisfaction with the ongoing cooperation with the FDA. She emphasized the urgent need for treatment options for Galactosemia, a progressive disease, and highlighted that the potential approval of govorestat could significantly impact patients and their families. Shendelman also discussed the company's efforts to ensure commercial readiness, which includes establishing an effective patient access program, increasing physician awareness, and engaging with payors.

Galactosemia is a rare genetic metabolic disorder that inhibits the proper metabolism of galactose, a simple sugar found in foods and produced by the body. Failure to metabolize galactose correctly leads to the formation of galactitol, a toxic metabolite that causes various neurological issues, including speech, cognition, behavior, and motor skills deficiencies. Additionally, it can result in juvenile cataracts and ovarian insufficiency in women. The disease affects approximately 3,000 individuals in the United States and sees about 80 new cases each year. In the European Union, around 4,000 people are affected, with approximately 120 new cases annually. Newborns in the US and most EU countries are screened for Galactosemia, allowing for early identification and intervention.

Govorestat (AT-007) is an Aldose Reductase Inhibitor (ARI) that penetrates the central nervous system and is being developed to treat several rare neurological conditions, including Galactosemia, SORD Deficiency, and PMM2-CDG. Clinical trials have shown that govorestat provides benefits in daily living activities, behavioral symptoms, cognition, fine motor skills, and tremor in children aged 2-17 with Galactosemia. It also significantly reduces plasma galactitol levels in both children and adults, addressing the primary cause of tissue damage and long-term complications in Galactosemia.

The Phase 3 INSPIRE trial for SORD Deficiency patients revealed that govorestat significantly reduced blood sorbitol levels over a 12-month period compared to a placebo. There was also a notable correlation between reduced sorbitol levels and improvements in clinical outcomes such as walking tests and sensory function. Furthermore, govorestat showed significant effects on the CMT Health Index (CMT-HI), benefiting lower limb function, mobility, fatigue, pain, sensory function, and upper limb function.

Govorestat has received several designations from regulatory bodies, including Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and SORD Deficiency. The U.S. Food and Drug Administration (FDA) has granted it Orphan Drug Designation for treating Galactosemia, PMM2-CDG, and SORD Deficiency, as well as Pediatric Rare Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.

Applied Therapeutics is pushing forward with its mission to develop innovative treatments for rare and high unmet medical needs. In addition to govorestat, the company is developing AT-001 for Diabetic Cardiomyopathy and AT-003 for Diabetic Retinopathy. Their comprehensive approach aims to address significant gaps in current treatment options for these severe conditions.