Applied Therapeutics is moving forward with regulatory submissions for govorestat (AT-007) in both the U.S. and Europe for the treatment of classic Galactosemia, a rare genetic metabolic disease. The company is scheduled to meet with the FDA this summer to discuss a potential New Drug Application (NDA) submission, while plans are underway to submit a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) in the fall.
Regulatory Milestones for Govorestat
The FDA has granted Applied Therapeutics a pre-NDA meeting to discuss the potential submission for govorestat. The company believes that existing clinical efficacy data, galactitol biomarker results, and a favorable safety profile could support an NDA submission. Applied Therapeutics aims to align with the FDA on the specifics of the submission and plans to submit the NDA in the fall, pending a positive outcome from the meeting.
In Europe, Applied Therapeutics and its commercial partner, Advanz Pharma, have engaged with EMA rapporteurs and are preparing to submit an MAA. The submission is planned for the fall to allow sufficient time for the approval of the Pediatric Investigational Plan (PIP) and to incorporate feedback from the EMA meeting.
Clinical Evidence and Galactosemia Treatment
Govorestat is a CNS-penetrant Aldose Reductase Inhibitor (ARI) being developed for rare neurological diseases, including Galactosemia. Clinical studies in children aged 2-17 with Galactosemia have demonstrated that govorestat provides clinical benefits in activities of daily living, behavior, cognition, fine motor skills, and tremor. The treatment also significantly reduced plasma galactitol levels in both adult and pediatric patients. Galactitol is a toxic metabolite that contributes to tissue damage and long-term complications in Galactosemia.
Unmet Needs in Galactosemia
Galactosemia is a rare genetic disorder where the body cannot properly metabolize galactose, leading to the accumulation of galactitol. This accumulation results in neurological complications, including speech and cognitive deficiencies, behavioral issues, motor skill impairments, juvenile cataracts, and ovarian insufficiency in females. Despite dietary restrictions, endogenous galactose production continues to cause toxic metabolite buildup. Currently, there are no approved treatments for Galactosemia, highlighting the significant unmet medical need.
Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics, stated, "We believe that govorestat offers compelling efficacy alongside a favorable safety profile and represents a transformative treatment option for patients with Galactosemia... There are currently no approved treatments for Galactosemia, and we hope to bring govorestat to patients as the first treatment for Galactosemia as soon as possible."
About Galactosemia
Galactosemia affects approximately 3,000 patients in the U.S. with 80 new births per year, and about 4,000 patients in the EU with 120 new births per year. Govorestat has received Orphan Medicinal Product Designation from the EMA for both Galactosemia and SORD Deficiency, as well as Orphan Drug Designation and Pediatric Rare Disease designation from the FDA for Galactosemia.
