EU CHMP issues negative opinion on Apellis MAA application for pegcetacoplan (Syfovre) injection for GA
Apellis will seek re-examination from the CHMP following the opinion, according to a news release.
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The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has again recommended the refusal of marketing authorisation for Syfovre (pegcetacoplan), according to a press release from Apellis Pharmaceuticals.1 The first negative marketing authorisation application (MAA) opinion came earlier this year in January. At the time, Apellis stated it was expecting the negative outcome based on a “negative trend vote following an oral explanation meeting in December 2023.”
The most recent refusal was issued by the CHMP2 with the explanation that, while Syfovre did slow the growth of geographic atrophy (GA) lesions, it “did not lead to clinically meaningful benefits for patients.” The committee further explained by stating, “It was noted 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.” Because of this, the CMHP stated “a positive balance of benefits and risks of Syfovre in the treatment of geographic atrophy caused by AMD could not be established” and therefore recommended a rejection.
The studies mentioned in the statement from the CHMP were the OAKS and DERBY multi-centre, 2-year, Phase 3, randomised, double-masked trials.
Apellis stated it plans on seeking re-examination of pegcetacoplan3 and a final opinion is expected in the fourth quarter of 2024. The company noted there were "multiple dissenting votes by CHMP members" in regards to the issued MAA opinion. Furthermore, an Ad Hoc Expert Group meeting took place in which experts agreed that size of GA lesion is an acceptable primary outcome measure for a trial in GA and that microperimetry is the best available functional measure in GA.
“We remain steadfast in our commitment to GA patients in Europe who have no treatment for this devastating disease, which leads to irreversible vision loss,” said Jeffrey Eisele, PhD, chief development officer at Apellis. “It is encouraging to see the broad support for pegcetacoplan within the European retina community, and we are focused on promptly initiating the re-examination to bring this important treatment to those in need.”
Syfovre was approved by the US FDA in February of 2023 to treat GA secondary to AMD.
References:
Apellis Announces Negative CHMP Opinion for Pegcetacoplan for GA in the European Union and Plans to Seek Re-Examination of Application. Press Release. Published 26 January, 2024. Accessed 1 July, 2024. https://investors.apellis.com/news-releases/news-release-details/apellis-announces-negative-chmp-opinion-pegcetacoplan-ga
Refusal of the marketing authorisation for Syfovre (pegcetacoplan). June 28, 2024. Accessed 1 July, 2024. https://www.ema.europa.eu/en/documents/smop-initial/questions-answers-refusal-marketing-authorisation-syfovre_en.pdf
Apellis Plans to Seek Re-Examination Following Negative CHMP Opinion for Pegcetacoplan for Geographic Atrophy (GA) in the EU. Published 28 June, 2024. Accessed 1 July, 2024. https://investors.apellis.com/news-releases/news-release-details/apellis-plans-seek-re-examination-following-negative-chmp
