Apellis provides updates on injection kits and rare safety events with Synfovre

Recommended use of filter needles included in certain injection kits: As part of the comprehensive investigation into the real-world safety events, internal structural variations were identified in the specific 19-gauge x 1½ inch filter needle included in certain injection kits. Filter needles are used to withdraw treatment from the vial when preparing for an injection procedure. A causal relationship has not been established between the structural variations in this 19-gauge filter needle and the rare events of retinal vasculitis in the real world. 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.

Update on rare events of retinal vasculitis reported to date: Caroline Baumal, M.D., chief medical officer of Apellis stated. “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. We believe Synfovre 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 Synfovre vials have been distributed in the real world and for administration in clinical trials. This includes: .i. Over 78,000 vials distributed since launch, including commercial vials shipped and sample vials distributed to physician practices. ii. Over 26,000 vials distributed in the third quarter to date. iii. Approximately 24,000 Synfovre 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.

The company reported one additional event of occlusive retinal vasculitis, which occurred in May, and was reported after its last communication on July 29.i. Two of the patients had their Synfovre injection in April, three in May, and three in June. ii. All events of retinal vasculitis were observed after the first injection of Synfovre.

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.