NDA submitted to FDA and MAA validated by EMA in
Submissions are based on positive results from Phase 3 registrational ACTION-Galactosemia Kids study demonstrating consistent long-term clinical outcomes benefit across a range of functional measures and favorable safety profile
“The submissions of both the NDA and MAA for govorestat are supported by rapid and sustained reduction in galactitol, which resulted in a meaningful benefit on clinical outcomes across pediatric patients, alongside a favorable safety profile,” said
“There are currently no treatments available for Galactosemia, a serious progressive disease affecting over 3,000 patients in the United States,” said
The NDA and MAA submission packages include clinical outcomes data from the Phase 3 registrational ACTION-Galactosemia Kids study in children age 2-17 with Galactosemia, the Phase 1/2 ACTION-Galactosemia study in adult patients with Galactosemia, and preclinical data. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review. The MAA has been validated and will move to review by the EMA’s Committee for Medicinal Products for Human Use (CHMP).
About Galactosemia
Galactosemia is a rare genetic metabolic disease resulting in an inability to metabolize the simple sugar galactose. Galactose is found in foods, but is also produced endogenously by the body. When not metabolized properly, galactose is converted to the toxic metabolite, galactitol, which causes neurological complications, including deficiencies in speech, cognition, behavior, and motor skills, and also results in juvenile cataracts and ovarian insufficiency (in women). There are approximately 3,000 patients with Galactosemia in the US and 80 new births per year, and approximately 4,000 patients with Galactosemia in the EU and 120 new births per year. Newborn screening for Galactosemia is mandatory in the US and most EU countries, leading to rapid identification of affected patients.
About Govorestat (AT-007)
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase inhibitor (ARI) in development for the treatment of several rare neurological diseases, including Galactosemia, SORD Deficiency, and PMM2-CDG.
In a study in children with Galactosemia aged 2-17, treatment with AT-007 demonstrated clinical benefit on activities of daily living, behavioral symptoms, cognition, fine motor skills and tremor. Govorestat also significantly reduced plasma galactitol levels in both adults and children with Galactosemia. Galactitol is a toxic metabolite responsible for tissue damage and long-term complications in Galactosemia.
Govorestat is also being studied in the ongoing Phase 3 INSPIRE trial, which is evaluating the effect of AT-007 vs. placebo in patients with SORD Deficiency on sorbitol reduction as well as clinical outcomes in approximately 50 patients aged 16-55 in the
Govorestat has received Orphan Medicinal Product Designation from the
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