Apellis Announces Plans to Submit 24-Month Phase 3 Data to the FDA for Pegcetacoplan NDA for Geographic Atrophy (GA)
Apellis Pharmaceuticals provided an update on its new drug application (NDA) for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
The company plans to submit the 24-month efficacy data from the phase 3 DERBY and OAKS studies as part of its application. As previously announced, these data showed robust and consistent effects with monthly and every-other-month pegcetacoplan treatment in both studies, according to Apellis. The 24-month safety data were previously submitted as part of the 120-day update. The submission is planned for November 2022 and will be a Major Amendment to the NDA, extending the review period by 3 months with an expected Prescription Drug User Fee Act (PDUFA) target action date in 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, Apellis. “We expect the submission will have minimal impact to the timing of the launch, which was originally planned for January. Assuming a February PDUFA date, we would be prepared to launch immediately following an approval.”
Dr. Francois continued, “We will work closely with the FDA as they complete their review of the pegcetacoplan NDA and look forward to bringing the first potential therapy to people living with GA, who currently have no approved treatment for this relentless and debilitating disease.”
Apellis said it remains on track to submit an EU marketing authorization application, which will also include the 24-month results, to the European Medicines Agency by the end of 2022.
About the Phase 3 OAKS and DERBY Studies
OAKS (n=621) and DERBY (n=637) are phase 3, multicenter, randomized, double-masked, sham-controlled studies comparing the efficacy and safety of pegcetacoplan with sham injections across a broad and representative population of patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The primary objective of the studies was to evaluate the efficacy of monthly and every-other-month pegcetacoplan in patients with GA assessed by change in the total area of GA lesions from baseline as measured by fundus autofluorescence at 12 months. Patients continued to receive masked treatment for 24 months.