Applied Therapeutics to Present Extended INSPIRE Trial Data for Govorestat in SORD Deficiency at PNS Annual Meeting
• Applied Therapeutics will present full 12-month results from the Phase 3 INSPIRE trial of govorestat for SORD Deficiency at the Peripheral Nerve Society 2025 Annual Meeting in Edinburgh, Scotland.
• The presentation will include new topline 18-month and 24-month data from the double-blind, placebo-controlled registrational trial, extending beyond the previously reported 12-month results.
• Govorestat, a CNS-penetrant Aldose Reductase Inhibitor, has received multiple regulatory designations including Orphan Drug status from both FDA and EMA for treating rare diseases including CMT-SORD.
FDA Rejects Applied Therapeutics' Govorestat for Classic Galactosemia, Citing Clinical Deficiencies
• The FDA issued a Complete Response Letter for Applied Therapeutics' govorestat, citing deficiencies in the clinical application for treating classic galactosemia.
• Govorestat, a CNS-penetrant aldose reductase inhibitor, aimed to reduce toxic galactitol levels and improve neurological complications in galactosemia patients.
• Applied Therapeutics plans to meet with the FDA to discuss resubmission or appeal, while continuing govorestat's development for SORD deficiency.
• The Galactosemia Foundation expressed disappointment, emphasizing the urgent need for treatments and advocating for reconsideration by the FDA.
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