New York-based Applied Therapeutics disclosed on Wednesday that the FDA has canceled an October advisory committee meeting for its potential treatment for the genetic disorder classic galactosemia, sending shares $APLT of the company up by more than 65%.
The announcement follows an FDA delay in April, when the agency said it needed additional time “to review supplemental analyses of previously submitted data,” according to the biotech.
A clinical study with children ages 2 to 17 who received govorestat missed its primary endpoint, but Applied Therapeutics pointed to improvements in behavior, tremors and other endpoints as evidence of efficacy. Biotech analysts at Leerink Partners and William Blair believe the treatment will be approved.
“Although the FDA’s decision to not host an AdCom meeting for govorestat is unexpected, we view this outcome as favorable for Applied as it suggests that the regulators are comfortable with the totality of the clinical data,” analysts at William Blair wrote on Wednesday. “After speaking with management, we highlight that the additional data the company previously submitted caused the sensitivity analysis for the primary endpoint to be positive. We suspect this data gave the agency greater confidence in govorestat’s efficacy and was a major contributor to the FDA’s decision to no longer hold an AdCom.”
If approved, govorestat would also come with a priority review voucher, which Applied could sell for about $100 million and further extend its cash runway.
“Collectively, we continue to think the galactosemia data are supportive of approval based on: the significant reduction in galactitol, the consistency of the data across several clinical endpoints, and the increasing separation of the govorestat group from placebo over time, especially in the context of a severe, rare disease with no other treatment options,” a Leerink analyst wrote Wednesday.