Two weeks after the American Society of Retina Specialists (ASRS) reported cases of occlusive and non-occlusive retinal vasculitis associated with the use of Apellis Pharmaceuticals, Inc.’s Syfovre (pegcetacoplan injection) 15 mg/0.1 mL, the company has provided an update on its review.
Refresh me on Syfovre.
Approved by the FDA in February 2023, Syfovre is currently the first and only therapy for geographic atrophy secondary to age-related macular degeneration (AMD).
Via intravitreal injection, it works as a C3 inhibition therapy to regulate excessive activation of the complement cascade (part of the immune system), which could lead to the onset and progression of diseases such as GA.
And what did ASRS report?
The ASRS Research and Safety in Therapeutics (ReST) Committee notified ASRS members on July 15, 2023, that physicians have reported intraocular inflammation (IOI) in patients following a first injection of Syfovre, including mild-to-moderate and severe IOI as well as cases of retinal vasculitis and occlusive retinal vasculitis.
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Any changes in the number of reported cases?
The ASRS initially reported six cases; the latest update brings that total to seven confirmed cases of retinal vasculitis: four occlusive and three non-occlusive.
“Two of these events followed injections in April (2023), two in May (2023), and three in June (2023),” the company stated.
One other potential case is currently being evaluated and has not yet been confirmed by Apeillis.
How many vials/injections had been distributed?
Per Apellis—and as of July 28, 2023—more than 68,000 vials have been commercially distributed and 23,000 clinical trial injections to date since Syfovre’s approval.
How’d the company respond?
An Apellis spokesperson told Glance on July 18, 2023, that Apellis was “thoroughly investigating all cases with external experts, including real-world procedure techniques and potential risk factors.”
And its latest update?
According to Apellis CEO and Co-Founder Cedric Francois, MD, PhD, the company’s ongoing review of the Syfovre has so far resulted in “no indication that drug product or manufacturing issues contributed to these events.”
He added that, “these events continue to be very rare.”
What else?
The company also reiterated that, after an ongoing secondary review, there were no new safety findings identified in the phase 3 clinical trials and no changes in the product’s formation between the trials and commercial distribution.
Give me specifics.
Based on the review, Apellis reported:
- No manufacturing-related issues impacting product quality
- No quality issue and no contaminants
- No single manufacturing lot implicated
- No indication of drug-related immunogenicity (in clinical trial data)
- No reports of retinal vasculitis in phase 3 clinical trials (based on review of all reported IOI cases)
And their next steps?
Per the spokesperson, the company is in the process of “thoroughly investigating all cases with external experts, including real-world procedure technique and potential risk factors.”
They also stated that, “patient safety is our top priority, and we take any (AEs) seriously.”
Any other news on the Syfovre front?
Actually, yes.
At the annual ASRS 2023 meeting, the company also just released promising data on the drug from its GALE extension study (NCT04770545) that followed 30 months of continuous treatment with Syfovre.
Go on …
The study reinforced long-term efficacy and safety for Syfovre as both a monthly and every-other-month treatment.
Apellis CMO Caroline Baumal, MD expressed the company’s enthusiasm for the data that found Syfovre, “reduced GA lesion growth by up to 45% in patients with nonsubfoveal lesions.”