Apellis Provides Updates on Injection Kits and Rare Safety Events with GA Drug Syfovre
Shares of Apellis Pharmaceuticals are soaring on Wednesday after the company identified a potential cause of the rare events of retinal vasculitis reported in real-world use of its recently approved GA treatment Syfovre.
As part of the investigation into the safety events, 'internal structural variations' were identified in the specific 19-gauge x 1½ inch filter needle included in certain injection kits. A causal relationship has not been established between the structural variations in the 19-gauge filter needles, which are used to withdraw treatment from the vial when preparing for an injection procedure, and the rare events of retinal vasculitis in the real world, according to Apellis.
The company recommends that practitioners immediately discontinue use of any injection kits that contain the 19-gauge filter needle and use injection kits with the 18-gauge filter needle, which are already in distribution. While injection kits previously contained one of two types of filter needles (either 18- or 19-gauge), Apellis is now exclusively distributing injection kits with the 18-gauge filter needle.
“Based on the findings from our investigation, we believe it is prudent that practitioners only use the kits with the 18-gauge filter needle, which are already in distribution. This recommendation is out of an abundance of caution as patient safety is our top priority,” Caroline Baumal, MD, chief medical officer of Apellis, said in a company news release.
Apellis received FDA approval for Syfovre, a targeted C3 therapy, in February, becoming the first FDA approved drug for GA. However, in July, a notification from the ASRS Research and Safety in Therapeutics (ReST) Committee alerted members to rare cases of retinal vasculitis that occurred after the first injection of Syfovre. The safety concerns, posted online to ASRS members, caused many clinicians to halt use of the drug until more information was released, and ultimately caused Apellis’s shares to lose about two-thirds of their value.
This week, Apellis provided an update on the rare events of retinal vasculitis reported to date.
“To date, more than 100,000 vials have been distributed for commercial use and for administration in clinical trials, and the events of retinal vasculitis continue to be very rare at an estimated real-world rate of 0.01% per injection," Dr. Baumal said. "We believe Syfovre is an important medicine for patients living with this chronic disease and are committed to providing patients with a meaningful and safe treatment.
Over 100,000 Syfovre vials have been distributed in the real world and for administration in clinical trials. This includes:
Over 78,000 vials distributed since launch, including commercial vials shipped and sample vials distributed to physician practices. Over 26,000 vials distributed in the third quarter to date
Approximately 24,000 Syfovre injections administered in clinical trials to date
In total, eight events of retinal vasculitis (five occlusive, three non-occlusive) have been confirmed. The last confirmed event of retinal vasculitis occurred on June 20, based on a review of adverse events reported to the company
This includes one additional event of occlusive retinal vasculitis, which occurred in May, and was reported after the company's last communication on July 29
Two of the patients had their Syfovre injection in April, three in May, and three in June
All events of retinal vasculitis were observed after the first injection of Syfovre
One patient remained stable at baseline vision, two patients have recovered vision nearly back to baseline, two patients have severe vision impairment which is unlikely to be resolved, and three patients’ outcomes are still pending
There are two events of suspected retinal vasculitis. As previously disclosed, there was one event that occurred in May and the patient’s vision has returned to baseline. The other event occurred in August and the patient’s outcome is pending. Neither event has been confirmed.
All post-marketing adverse events reported to the company, including events of retinal vasculitis, are reviewed by Apellis’ Medical and Safety Committee. Any suspected events of vasculitis are also evaluated by external retina/uveitis specialists for adjudication.