CHMP recommends refusal of approval for Syfovre (pegcetacoplan) to treat geographic atrophy caused by age-related macular degeneration. Apellis Pharma
The European Medicines Agency has recommended the refusal of the marketing authorisation for Syfovre, a medicine intended for the treatment of geographic atrophy caused by age-related macular degeneration (AMD).
AMD is a disease that affects the central part of the retina (called the macula) at the back of the eye. Geographic atrophy is an advanced form of AMD in which lesions (areas of cell death) form in the retina and macula, leading to loss of vision.
Although the studies showed that Syfovre slowed the growth of geographic atrophy lesions, this did not lead to clinically meaningful benefits for patients. 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. Therefore, the Agency’s opinion was that the benefits of Syfovre did not outweigh its risks and it recommended refusing marketing authorisation.
Apellis plans to seek immediate re-examination of its application.
The MAA is based on results from the Phase III 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 III analyses that have been presented at medical congresses.
See- The Lancet.VOLUME 402, ISSUE 10411, P1434-1448, OCTOBER 21, 2023."Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase III trials.": Jeffrey S Heier, MD ,Eleonora M Lad, MD ,Prof Frank G Holz, MD, Prof Philip J Rosenfeld, MD, Prof Robyn H Guymer, MBBS David Boyer, MD, et al. Published:October 21, 2023DOI:https://doi.org/10.1016/S0140-6736(23)01520-9.
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