After pushing back the decision date for Applied Therapeutics’ metabolic disorder drug govorestat, the FDA has now decided that a planned advisory committee meeting won’t be required.
The agency had originally expected an approval decision for the aldose reductase inhibitor for the end of August, but, by March, the FDA had bumped this back three months to Nov. 28. At the time, the regulator told Applied that more time was needed to examine supplemental analyses of already submitted data and determined that the additional info constitutes a major amendment to the new drug application.
Applied announced Wednesday morning that while the Nov. 28 deadline is still in place, the FDA had informed the biotech during a late-cycle review meeting that the advisory committee meeting to discuss the application—which had been penciled in for Oct. 9—is no longer required.
“We are incredibly pleased by the ongoing collaborative dialogue with the FDA during the NDA review process, and we look forward to continuing to work together with the agency to bring the first potential treatment to classic galactosemia patients,” Applied’s CEO Shoshana Shendelman, Ph.D., said.
“Our commitment to the cassic galactosemia community is further supported by our thoughtful commercial preparation, focused on establishing an effective patient access program, high physician awareness and strong payor engagement,” Shendelman added.
While analysts at William Blair said the FDA's decision was “unexpected,” they branded it as good news.
“We view this outcome as favorable for Applied as it suggests that the regulators are comfortable with the totality of the clinical data submitted to make a regulatory decision on or prior to the November 28 PDUFA,” the analysts said in a Sept. 18 note.
Applied’s confidence in govorestat has survived a phase 3 trial last year that showed the drug was no better than placebo at improving a composite of four measures—including language skills, self-care capabilities and more—among children with galactosemia. The rare disease can cause developmental delays, speech problems and motor function abnormalities.
Despite the failure, the New York-based biotech argued at the time that the data showed “consistent and sustained clinical benefit on activities of daily living, behavioral symptoms, cognition, adaptive behavior and tremor” and went ahead with filing a new drug application with the FDA.
Applied had planned to ask for U.S. approval on the strength of biomarker data, only for the FDA to say it would likely need evidence the drug candidate improves clinical outcomes to receive a positive decision. The phase 3 trial gave Applied evidence of the effect of govorestat, also known as AT-007, on clinical outcomes.