Apellis Expects to Receive a Negative Vote for its European Application for Pegcetacoplan for GA

Shares of Apellis Pharmaceuticals were significantly lower on Thursday after the company said it expects the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to adopt a negative opinion for intravitreal pegcetacoplan for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The vote is expected at CHMP's next meeting, which is scheduled to be held January 22-25, 2024.

Following the oral explanation meeting on December 13, Apellis said it was informed of a negative trend vote on the MAA for pegcetacoplan. If a negative opinion is adopted, Apellis said it plans to appeal the outcome and seek re-examination of the opinion.

“We are very disappointed in this outcome and saddened for the millions of GA patients in Europe who have no treatment for this leading cause of blindness,” Jeffrey Eisele, PhD, chief development officer, Apellis, said in a company news release. “We firmly believe in the strength of the phase 3 results, and we will continue working closely with the CHMP, the retina community, and patient advocacy groups with the goal of bringing pegcetacoplan to GA patients in Europe.”

The MAA is based on results from the phase 3 OAKS and DERBY studies at 24 months, which were published in The Lancet. Treatment with both every-other-month and monthly pegcetacoplan reduced GA lesion growth with increasing treatment effects over time and showed a well-demonstrated safety profile. Additionally, pegcetacoplan preserved visual function longer in multiple post hoc phase 3 analyses that have been presented at medical congresses, according to Apellis. 

Apellis received FDA approval for Syfovre, a targeted C3 therapy, in February, becoming the first FDA approved drug for geopgraphic atrophy (GA). However, in July, a notification from the ASRS Research and Safety in Therapeutics (ReST) Committee alerted members to rare cases of retinal vasculitis that occurred after the first injection of Syfovre. In August, the company identified a potential cause of the rare events of retinal vasculitis reported in real-world use of Syfovre. Apellis said 'internal structural variations' were identified in the specific 19-gauge by 1½ inch filter needle included in certain injection kits.

More than 2.5 million people in Europe are living with GA,¹ a progressive and irreversible form of vision loss that severely impairs independence and quality of life.

References

¹Apellis data on file reflecting analysis of Colijin et al., 2016; Wong et al. 2014; Rudnicka et al., 2014; Korb et al., 2014; Piermarocchi et al., 2011; Fernandez-Arias et al., 2011; Augood et al., 2006.