EU CHMP confirms negative opinion on marketing authorization application of intravitreal pegcetacoplan from Apellis
The agency confirmed its June 2024 negative opinion after Apellis requested re-examination of EMA's initial opinion.
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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has confirmed its initial opinion to refuse marketing authorization for pegcetacoplan (Syfovre) for the treatment of geographic atrophy caused by age-related macular degeneration (AMD).
The initial opinion was issued June 27, 2024, after which Apellis sought re-examination of pegcetacoplan by the CHMP.
Cedric Francois, MD, PhD, CEO and co-founder of Apellis commented on the CHMP opinion in a press release from the company1 stating, “We are deeply disappointed by this outcome, which leaves millions of Europeans with GA without a treatment for this irreversible form of blindness. This CHMP opinion was made despite broad support for pegcetacoplan from the European retina community and multiple dissenting votes by CHMP members who supported a path to approval.”
Francois continued, stating “We have seen the profound impact this medicine has had for GA patients in the US and remain committed to expanding access to this crucial treatment, focusing on serving unmet patient needs in the U.S. and other regions globally.”
Syfovre was approved by the US FDA in February of 2023 to treat GA secondary to AMD.
Frank G. Holz, OAKS and DERBY study investigator and professor and chairman of the department of ophthalmology at the University of Bonn, Germany also commented on the opinion in the release from Apellis saying “It is disappointing to know that we will not be able to provide access to this treatment to GA patients in the EU. As an ophthalmologist and retina specialist, I have seen how patients with GA lose their ability to read, drive, and even see faces. The Phase 3 data for pegcetacoplan are clinically meaningful and showed the potential to make a difference for EU patients.”
Initial concerns stated by the CHMP regarding pegcetacoplan included that while Syfovre did slow the growth of GA lesions, it “did not lead to clinically meaningful benefits for patients.” Furthermore, the agency stated “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.”2
The agency stated that these concerns did not change after re-examination of data provided and consideration of information shared by patients and healthcare professional organizations. The agency noted that while it recognized the unmet medical need for effective treatment for patients with GA in the EU, the magnitude of Syfovre's effectiveness did not outweigh the potential risks.2
References:
Apellis Receives Negative CHMP Opinion for Pegcetacoplan for Geographic Atrophy (GA) in the EU Following Re-Examination. Press Release; September 20, 2024. Accessed September 25, 2024. https://investors.apellis.com/news-releases/news-release-details/apellis-receives-negative-chmp-opinion-pegcetacoplan-geographic
Refusal of the marketing authorisation for Syfovre (pegcetacoplan). September 19, 2024. Accessed September 25, 2024. https://www.ema.europa.eu/en/documents/smop-ini
