The US Food and Drug Administration (FDA) has announced a three-month extension to the review period for Applied Therapeutics‘ New Drug Application (NDA) for govorestat (AT-007), a potential treatment for classic galactosemia, a rare genetic metabolic disease.
The FDA has requested more time to review supplemental analyses of priorly submitted data, which has been classified as a major amendment to the NDA.
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Applied Therapeutics submitted this data in response to the regulator’s routine information requests.
A decision on the approval for the asset under the Prescription Drug User Fee Act (PDUFA) is now expected by 28 November this year.
The company obtained priority review designation for the NDA of govorestat last month.
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The product has also been granted pediatric rare disease designation and is eligible for a Priority Review Voucher (PRV) upon approval.
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By GlobalDataAn investigational aldose reductase inhibitor (ARI), govorestat is being developed for treating several rare diseases.
The NDA is supported by data demonstrating a quick and lasting decline in galactitol levels, which has shown meaningful clinical outcomes in paediatric patients, along with a favourable safety profile.
The submission included clinical data from the Phase III ACTION-Galactosemia Kids study in kids aged two to 17 years; the Phase I/II ACTION-Galactosemia study in adults; as well as preclinical findings.
If approved, govorestat would become the first medication indicated for the treatment of galactosemia and aid Applied Therapeutics to enter the commercial product market.
Additionally, the company has filed a marketing authorization application (MAA) with the European Medicines Agency (EMA) to treat classic galactosemia. The MAA was validated by the EMA in December last year.
The EMA’s Committee for Medicinal Products for Human Use (CHMP) is currently reviewing the application, with a decision expected in the fourth quarter of this year.
Applied Therapeutics founder and CEO Shoshana Shendelman said: “While the PDUFA action date extension represents a delay, we remain confident in the potential for govorestat approval for Galactosemia and we will continue to work closely with the FDA throughout the review process.
“During this time, we are committed to maintaining the expanded access programme for govorestat to ensure patients with Galactosemia have the opportunity to receive this important treatment.”
