Applied Therapeutics' Govorestat Nears Potential Approval for Galactosemia and SORD Deficiency
Key Insights
Applied Therapeutics anticipates FDA decision on govorestat for Classic Galactosemia (search) by November 28, 2024, and EMA decision in Q1 2025.
NDA submission for govorestat to treat SORD Deficiency (search) under Accelerated Approval is expected in early Q1 2025.
Govorestat's NDA and MAA submissions are supported by clinical data demonstrating galactitol reduction and improved outcomes in pediatric patients.
Applied Therapeutics is on track to potentially secure its first commercial product with govorestat, a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI), for the treatment of Classic Galactosemia (search) and SORD Deficiency (search). The company's New Drug Application (NDA) for govorestat in Classic Galactosemia is under Priority Review by the FDA, with a Prescription Drug User Fee Act (PDUFA) target action date of November 28, 2024. Concurrently, the Marketing Authorization Application (MAA) is under review by the European Medicines Agency (EMA), with a decision expected in the first quarter of 2025.
Regulatory Milestones and Clinical Data
The NDA and MAA submissions for govorestat are supported by data from the Phase 3 ACTION-Galactosemia (search) Kids study, a registrational trial in children aged 2-17 with Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients, and preclinical data. These data packages highlight the rapid and sustained reduction in galactitol levels achieved with govorestat, which correlated with meaningful improvements in clinical outcomes and a favorable safety profile. If approved, govorestat would be the first medication indicated for the treatment of Galactosemia.
Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics, stated, "We are proud of the significant progress we’ve made this quarter as we prepare for a transformational year ahead, with a focus on transitioning from a clinical-stage company to a commercial organization... We remain confident in the promise of govorestat and its ability to address the underlying mechanisms of both diseases. We look forward to the opportunity to bring govorestat to patients in 2025."
SORD Deficiency Program
Applied Therapeutics also anticipates submitting an NDA for govorestat for the treatment of SORD Deficiency (search) under Accelerated Approval in early Q1 2025. This follows a Type C meeting with the FDA to align on the regulatory path forward. The review and potential approval of govorestat for SORD Deficiency are independent of the ongoing review for Classic Galactosemia (search). Patients in the Phase 3 INSPIRE study have transitioned to open-label govorestat treatment to generate additional safety data.
Financial Position
As of September 30, 2024, Applied Therapeutics reported cash, cash equivalents, and short-term investments totaling $98.9 million, compared to $49.9 million at the end of 2023. Research and development expenses for the third quarter of 2024 were $14.8 million, an increase from $10.8 million for the same period in 2023. The company's net loss for the third quarter of 2024 was $68.6 million, or $0.48 per share, compared to a net loss of $42.4 million, or $0.47 per share, for the third quarter 2023.
About Govorestat
Govorestat is designed to inhibit the aldose reductase enzyme, which is implicated in the pathogenesis of Galactosemia (search) and SORD Deficiency (search). By reducing the accumulation of toxic metabolites, govorestat aims to address the underlying mechanisms of these rare metabolic diseases.