Benzinga | Jan 3, 2022 07:01AM EST

Applied Therapeutics to Hold on Submitting a New Drug Application for its AT-007 for Treatment of Galactosemia Pending Additional Discussions with the FDA

The ongoing ACTION-Galactosemia Kids Phase 3 study is evaluating the impact of AT-007 treatment vs. placebo on clinical outcomes over time, including cognition, speech, behavior and motor skills. Clinical outcomes are assessed every 6 months by a firewalled committee. The first assessment will be completed in the first quarter of 2022, and then every 6 months thereafter until the study reaches statistical significance.

"While disappointed by this change in direction by the FDA, we remain committed to bringing this important treatment to patients with Galactosemia," said Shoshana Shendelman, PhD, CEO, Founder and Chair of the Board of Applied Therapeutics. "We will continue to work with the FDA to determine the most expeditious path forward to regulatory approval and will provide an update on timing and plans accordingly."