December 2023 JAMA Ophthalmology Viewpoint Piece About Syfovre Calls for Independent and Unbiased Evaluation
Since the February 2023 FDA approval of Syfovre (pegcetacoplan), Apellis Pharmaceuticals has had to contend with various Syfovre safety issues related to its eye injection treatment for geographic atrophy. In our December 4, 2023 post, "Syfovre Update: New Warnings About Retinal Vasculitis and/or Retinal Vascular Occlusion After Syfovre Injections", we reported on the November 2023 Syfovre label change that came after numerous cases of eye inflammation and severe vision loss.
A Viewpoint piece published December 7, 2023, online by JAMA Ophthalmology, "Concerning Syfovre Approval for Geographic Atrophy", touches upon these Syfovre safety issues. It begins as follows:
Syfovre (pegcetacoplan) was recently approved by the US Food and Drug Administration (FDA) as a treatment to slow the rate of geographic atrophy (GA) growth, although not as a treatment for visual acuity. Though there is much hope and marketing about tissue preservation with this drug, to date, there are substantial safety and efficacy concerns that might give clinicians pause before widespread adoption.
That December 2023 JAMA Ophthalmology Viewpoint piece was reported on by Peter Wehrwein, Managing Editor of Managed Healthcare Executive, in his December 7, 2023 article, "Independent Assessment of Syfovre Needed, Argues JAMA Ophthalmologist Viewpoint":
An independent panel of retinal specialists and statisticians should assess the clinical trial data for Syfovre (pegcetacoplan injection) and provide an unbiased evaluation of the drug in light of the lack of peer-review publication and a complete FDA review, argues Ira Schachar, M.D., M.Sc., the author of an opinion piece published today in JAMA Ophthalmology.
Schachar also suggests that widespread use of Syfovre should wait until there is a complete FDA scientific analysis of the drug or a peer-reviewed publication of the study results. He says the data released by Apellis Pharmaceuticals raises methodological questions and he questions whether “repeated measures and missing data have been appropriately handled.” Schachar, who was an assistant professor at Stanford prior to joining the North Bay group, says in the one-page viewpoint piece that questions about the Syfovre statistics are “particularly important” because of the high dropout rates from two important, phase 3 trials for the drug called OAKS and DERBY.
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In a December 9, 2023 Editor’s Note by Neil M. Bressler, MD, Editor in Chief of the JAMA Ophthalmology medical journal, published as a Comment to "Concerning Syfovre Approval for Geographic Atrophy", it is pointed out:
Since the submission, revision, and acceptance of this Viewpoint [by Dr. Schachar], the primary outcome results of two 24-month, multicenter randomized clinical trials to assess the efficacy and safety of [Syfovre (pegcetacoplan)] compared with sham treatment in patients with geographic atrophy were published.... This information was not publicly available at the time of acceptance of this Viewpoint. A formal publication to address this should be published in JAMA Ophthalmology by early 2024.
The results of those two clinical trials referred to therein, OAKS and DERBY, were reported in this medical journal article, "Pegcetacoplan for the treatment of geographic atrophy secondary to age-related macular degeneration (OAKS and DERBY): two multicentre, randomised, double-masked, sham-controlled, phase 3 trials", published October 21, 2023, by The Lancet.
In the Interpretation part of the Abstract for this Lancet medical journal article, lead author Jeffrey S. Heier, M.D., of Ophthalmic Consultant of Boston, and his colleagues stated:
[Syfovre (pegcetacoplan)], the first treatment approved by the US Food and Drug Administration for geographic atrophy, slowed geographic atrophy lesion growth with an acceptable safety profile.
The OAKS and DERBY trials were funded by Apellis Pharmaceuticals.
We will continue to monitor the various Syfovre safety issues and report further significant developments here on our Drug Injury Watch website.