FDA to Decide on Govorestat for Classic Galactosemia by November 28 After Advisory Committee Not Required
核心洞察
The FDA has completed its late-cycle review meeting for govorestat, a treatment for Classic Galactosemia (搜索), and an advisory committee meeting will not be required.
The Prescription Drug User Fee Act (PDUFA) target action date for govorestat remains on track for November 28, 2024, indicating a decision is expected by then.
Govorestat, a CNS penetrant Aldose Reductase (搜索) Inhibitor, has shown clinical benefits in children with Galactosemia (搜索), improving daily living activities and reducing plasma galactitol levels.
Applied Therapeutics announced that the FDA has completed its late-cycle review meeting for the New Drug Application (NDA) of govorestat for Classic Galactosemia (搜索) and has indicated that an Advisory Committee meeting, previously scheduled for October 9, 2024, will no longer be required. The PDUFA target action date remains on track for November 28, 2024.
Shoshana Shendelman, PhD, Founder and CEO of Applied Therapeutics, expressed satisfaction with the ongoing dialogue with the FDA and the potential of govorestat to transform the lives of patients and families affected by this serious disease.
About Galactosemia
Classic Galactosemia (搜索) is a rare genetic metabolic disorder characterized by the body's inability to properly metabolize galactose, a simple sugar found in food and produced endogenously. The improper metabolism of galactose leads to the accumulation of galactitol, a toxic metabolite, which can cause neurological complications such as speech deficiencies, cognitive impairment, behavioral issues, motor skill deficits, juvenile cataracts, and ovarian insufficiency in females. In the US, approximately 3,000 individuals are affected by Galactosemia, with 80 new births per year. In the EU, there are around 4,000 patients and 120 new births annually. Newborn screening for Galactosemia is mandatory in the US and most EU countries.
Govorestat (AT-007) as a Potential Treatment
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase (搜索) Inhibitor (ARI) being developed to treat rare neurological diseases, including Galactosemia (搜索). Clinical studies in children with Galactosemia (ages 2-17) have demonstrated that govorestat provides clinical benefits in activities of daily living, behavioral symptoms, cognition, fine motor skills, and tremor. The treatment has also been shown to significantly reduce plasma galactitol levels in both adults and children with Galactosemia.
In the Phase 3 INSPIRE trial involving patients with SORD Deficiency (搜索), an interim analysis at 12 months showed a statistically significant reduction in blood sorbitol levels with govorestat treatment compared to placebo (p<0.05). The study also demonstrated a statistically significant correlation between sorbitol levels and changes in clinical outcomes, such as the 10-meter walk run test, dorsiflexion, and 6-minute walk test (p<0.05). Furthermore, govorestat exhibited highly statistically significant effects on the CMT Health Index (CMT-HI), a patient-reported outcome measure, indicating benefits in lower limb function, mobility, fatigue, pain, sensory function, and upper limb function (p<0.01).
Govorestat has been granted Orphan Medicinal Product Designation by the European Medicines Agency (EMA) for both Galactosemia (搜索) and SORD Deficiency (搜索). It has also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia, PMM2-CDG (搜索), and SORD Deficiency, as well as Pediatric Rare Disease designation for Galactosemia and PMM2-CDG, and Fast Track designation for Galactosemia.
Applied Therapeutics is focused on establishing an effective patient access program, increasing physician awareness, and ensuring strong payor engagement in anticipation of potential approval.