Applied Therapeutics' Govorestat Awaits Potential FDA Approval for Classic Galactosemia

Applied Therapeutics查看公司主页 is on track for a potentially transformative year as it awaits a decision from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for govorestat查看药品详情 in the treatment of Classic Galactosemia (搜索)搜索该疾病. The FDA's decision is expected by the Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024. Simultaneously, the company is preparing an NDA submission for govorestat to treat SORD Deficiency (搜索)搜索该疾病, anticipated in early Q1 2025.

Regulatory Milestones for Govorestat

The NDA for govorestat查看药品详情 in Classic Galactosemia (搜索)搜索该疾病 is currently under review within the FDA's Division of Rare Diseases and Medical Genetics. Govorestat has been granted Pediatric Rare Disease designation, which would qualify Applied Therapeutics查看公司主页 for a Priority Review Voucher (PRV) upon approval. In parallel, the Marketing Authorization Application (MAA) for govorestat in Classic Galactosemia is under review by the European Medicines Agency (EMA), with a decision expected in early Q1 2025. The NDA and MAA submissions are supported by data demonstrating rapid and sustained reduction in galactitol, leading to meaningful clinical benefits in pediatric patients, along with a favorable safety profile. These submissions include clinical outcomes data from the Phase 3 ACTION-Galactosemia Kids study in children aged 2-17, the Phase 1/2 ACTION-Galactosemia study in adult patients, and preclinical data.

Following a Type C meeting with the FDA, Applied Therapeutics查看公司主页 plans to submit an NDA under Accelerated Approval for govorestat查看药品详情 in SORD Deficiency (搜索)搜索该疾病 in early Q1 2025. The review and potential approval of govorestat for SORD Deficiency are independent of the ongoing review for Classic Galactosemia (搜索)搜索该疾病. If govorestat is approved for Classic Galactosemia, the SORD Deficiency submission will be a supplementary New Drug Application (sNDA). Patients from the Phase 3 INSPIRE study have transitioned to open-label govorestat treatment for continued safety data collection.

Clinical Data Presentations

Applied Therapeutics查看公司主页 presented clinical data and development characterization of govorestat查看药品详情 for Classic Galactosemia (搜索)搜索该疾病 at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium and the American Society of Human Genetics (ASHG) Annual Meeting 2024. These presentations highlighted the mechanism of disease pathogenesis in Classic Galactosemia, the design of the first clinical outcomes study in Classic Galactosemia, and the results of the ACTION-Galactosemia Kids study.

Financial Position

As of September 30, 2024, Applied Therapeutics查看公司主页 reported $98.9 million in cash and cash equivalents and short-term investments, compared to $49.9 million at December 31, 2023. Research and development expenses for the three months ended September 30, 2024, were $14.8 million, compared to $10.8 million for the same period in 2023. General and administrative expenses were $15.0 million for the three months ended September 30, 2024, compared to $4.7 million for the same period in 2023. The net loss for the third quarter of 2024 was $68.6 million, or $0.48 per basic and diluted common share, compared to a net loss of $42.4 million, or $0.47 per basic and diluted common share, for the third quarter 2023.

Applied Therapeutics查看公司主页 is focused on developing novel drug candidates against validated molecular targets in indications of high unmet medical need. Govorestat查看药品详情, a central nervous system penetrant Aldose Reductase (搜索)搜索该靶点 Inhibitor (ARI), is being developed for CNS rare metabolic diseases, including Galactosemia (搜索)搜索该疾病, SORD Deficiency (搜索)搜索该疾病, and PMM2-CDG (搜索)搜索该疾病. The company is also developing AT-001查看药品详情, a potent ARI, for Diabetic Cardiomyopathy (搜索)搜索该疾病 (DbCM), and AT-003查看药品详情, an ARI for Diabetic retinopathy (搜索)搜索该疾病.