EMA validates indication extension application of pegcetacoplan for treatment of kidney disease

The news of the extension follows CHMP confirmation of its refusal to grant marketing authorisation for Syfovre in the treatment of GA. Image credit: ©Crystal light – stock.adobe.com

Apellis Pharmaceuticals, Inc. and Sobi announced that the European Medicines Agency (EMA) has validated an indication extension application for pegcetacoplan (Aspaveli) for the treatment of two life-threatening kidney conditions. C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) are rare diseases, with no approved treatments until this point, according to the press release from Apellis.¹ In 2021, pegcetacoplan first received marketing authorisation from the European Commission for the treatment of a rare blood disorder.

The news of the extension follows two updates on pegcetacoplan in the ophthalmic space. In January, Apellis announced Australian approval of pegcetacoplan (Syfovre) for treatment of geographic atrophy (GA).² In September of 2024, the EMA’s Committee for Medicinal Products for Human Use (CHMP) issued confirmation of its initial refusal to grant marketing authorisation for Syfovre for the treatment of GA caused by age-related macular degeneration.³

The indication extension for treatment of C3G and Primary IC-MPGN reflects positive data¹ from the Phase 3 VALIANT study. The study findings demonstrated a statistically significant and clinically meaningful 68% (P <0.0001) proteinuria reduction in patients treated with pegcetacoplan. Additionally, the findings showed favorable safety and tolerability results.

The CHMP first issued a negative opinion on the marketing authorisation application for Syfovre in June 2024. Initial concerns stated by the CHMP regarding pegcetacoplan for treatment of GA noted that, while Syfovre did slow the growth of GA lesions, it “did not lead to clinically meaningful benefits for patients.”

At the time, the CHMP stated³ that “benefits of a treatment should impact patients’ everyday functioning, and this was not demonstrated in the studies. In terms of safety, regular injections into the eye carry a significant risk of adverse events, including the development of other forms of AMD or inflammation in the eye, that could further worsen vision.”

The Australian approval was based on results from the Phase 3 OAKS and DERBY studies. At 24 months, treatment with both every-other-month and monthly Syfovre was shown to slow GA progression and showed a generally well-tolerated safety profile.²

References

1. Apellis and Sobi Announce EMA Validation of Indication Extension Application for Aspaveli® (pegcetacoplan) for C3G and Primary IC-MPGN. Press release. Apellis Pharmaceuticals, Inc. Published February 20, 2025. Accessed February 20, 2025. https://investors.apellis.com/news-releases/news-release-details/apellis-and-sobi-announce-ema-validation-indication-extension

2. Harp MD. Apellis Pharmaceuticals receives Australian approval of pegcetacoplan (Syfovre) for treatment of geographic atrophy. Ophthalmology Times Europe. Published January 28, 2025. Accessed February 20, 2025. https://europe.ophthalmologytimes.com/view/apellis-pharmaceuticals-receives-australian-approval-of-pegcetacoplan-syfovre-for-treatment-of-geographic-atrophy

3. Harp MD. EU CHMP confirms negative opinion on marketing authorization application of intravitreal pegcetacoplan from Apellis. Ophthalmology Times. Published September 25, 2024. Accessed February 20, 2025. https://www.ophthalmologytimes.com/view/eu-chmp-confirms-negative-opinion-on-marketing-authorization-application-of-intravitreal-pegcetacoplan-from-apellis