The submission of 24-month efficacy data from DERBY and OAKS is classified as a Major Amendment to the New Drug Application, which delays the PDUFA target action date until February 2023.
Today, Apellis Pharmaceuticals announced plans to submit 24-month efficacy data from the Phase 3 DERBY and OAKS trials to the Food and Drug Administration (FDA) to strengthen their New Drug Application (NDA) for their lead candidate, pegcetacoplan, for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).1
Apellis believes that the 24-month efficacy data from DERBY and OAKS, which were released in August 2022, will strengthen the product profile at launch with small effects on the overall timeline. The submission is a Major Amendment to the NDA, which requires an extension to the review period. The PDUFA target action date is now February 2023.
“We look forward to sharing with the FDA the 24-month Phase 3 data, which demonstrate robust and increasing effects over time. We were able to complete the full 24-month data package faster than expected, providing us with an opportunity to submit these data prior to the November 26 PDUFA date. These longer-term data have the potential to provide an even stronger product profile at launch,” said Cedric Francois, MD, PhD, chief executive officer and co-founder of Apellis.
“We expect the submission will have minimal impact to the timing of the launch, which was originally planned for January,” Francois continues. “Assuming a February PDUFA date, we would be prepared to launch immediately following an approval.”
In both DERBY and OAKS, the data shows robust and consistent effects with monthly and every-other-month pegcetacoplan administration.2 If approved, pegcetacoplan will become the first therapy for the treatment of GA.
Despite the change in timeline in the United States, Apellis will forge forward with their plan to submit an EU marketing authorization application—now with the 24-month results—to the European Medicines Agency by the end of 2022.