28 March 2024 - The FDA has extended by three months the review period for Applied Therapeutics' New Drug Application (NDA) for govorestat (AT-007), a therapy for classical galactosemia.The FDA has established November 28, 2024 as the new effective date for the Prescription Drug User Fee Act (PDUFA).The company declared that the NDA for govorestat, a therapy for classical galactosemia, has been given priority evaluation by the FDA.
The Company declared in February 2024 that the FDA had accepted the NDA and given it priority review. Govorestat was previously designated as a pediatric uncommon condition, and if approved, it will be qualified to obtain a Priority Review Voucher (PRV).
Govorestat, if authorized, will be the first medication used to treat galactosemia and the first product that Applied Therapeutics sells commercially. Additionally, the business has applied for a Marketing Authorization Application (MAA) with the EMA to use govorestat to treat classical galactosemia. The MAA went into force in December 2023, and the EMA's Committee for Medicinal Products for Human Use is presently reviewing it (CHMP). The company anticipates hearing back from the EMA during 2024's fourth quarter.
The European Medicines Agency (EMA) has designated Govorestat as an orphan medication to treat SORD deficiency and galactosemia.Additionally, Govorestat has been granted fast track decisions for galactose blood disorders, pediatric rare disease designation for galactosemia and PMM2-CDG, and orphan drug status by the FDA for the treatment of galactosemia, PMM2-CDG, and SORD deficiency.
The quick and long-lasting reduction of galactitol, which produces notable improvements in clinical outcomes for pediatric patients with a good safety profile, supports the NDA application of govorestat. Preclinical data, a Phase 1/2 action-galactosemia trial in adult galactosemic patients, and clinical outcome data from a Phase 3 registration study of the disease in children aged 2-17 years are also included in the submission.
Additionally, governorestat was investigated in the ongoing Phase 3 INSPIRE research, which assessed the impact of AT-007 in comparison to placebo on sorbitol reduction and clinical outcomes in individuals with SORD deficiency. The investigation included about 50 patients, aged 16 to 55, from both Europe and the United States. Govorestat demonstrated a persistent, statistically significant decrease in sorbitol levels when compared to placebo at the 12-month interim analysis. It also achieved statistical significance on the pre-specified primary endpoint of a composite correlation of sorbitol with clinical outcomes. Furthermore, govorestat showed statistical significance on patient-reported outcome measures such as upper extremity function, lower extremities function, mobility, fatigue, discomfort, and sensory function on the CMT Health Index (CMT- hi).
