Regeneron Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) for aflibercept 8 mg for treatment of patients with wet age-related macular degeneration (wAMD), diabetic macular edema (DME) and diabetic retinopathy. The FDA target action date is June 27, 2023 following the use of a priority review voucher.
The BLA is supported by positive data from two pivotal trials – PULSAR in wAMD and PHOTON in DME.
In both trials, patients treated with aflibercept 8 mg (PULSAR n=673; PHOTON n=491) met the primary endpoint of non-inferiority in vision gains for both the 12- and 16-week dosing regimens after initial monthly doses at 48 weeks compared to patients treated with an EYLEA® (aflibercept) Injection (PULSAR n=336; PHOTON n=167) 8-week dosing regimen.
Additionally, the vast majority of patients randomized to aflibercept 8 mg in both trials were able to maintain the 12- and 16-week dosing regimens to which they were respectively initiated through 48 weeks (wAMD: 79% and 77%; DME: 91% and 89%).
The safety profile for aflibercept 8 mg was similar to EYLEA in both trials, and consistent with the known safety profile of EYLEA from previous clinical trials. Comparing aflibercept 8 mg to EYLEA, ocular adverse events occurred in 31% (n=491) versus 28% (n=167) in PHOTON and 38% (n=673) versus 39% (n=336) in PULSAR, and there were no cases of retinal vasculitis, occlusive retinitis or endophthalmitis in either trial.
Aflibercept 8 mg is being jointly developed by Regeneron and Bayer AG. In the U.S., Regeneron maintains exclusive rights to EYLEA and aflibercept 8 mg.
Bayer has licensed the exclusive marketing rights outside of the U.S., where the companies share equally the profits from sales of EYLEA and future sales of aflibercept 8 mg following any regulatory approvals.
Aflibercept 8 mg is investigational, and its safety and efficacy have not been evaluated by any regulatory authority.
About the aflibercept 8 mg trial program
PHOTON in DME and PULSAR in wAMD are double-masked, active-controlled pivotal trials that are being conducted in multiple centers globally. In both trials, patients were randomized into 3 treatment groups to receive either: aflibercept 8 mg every 12 weeks, aflibercept 8 mg every 16 weeks, or EYLEA every 8 weeks.
Patients treated with aflibercept 8 mg in both trials had 3 initial monthly doses, and patients treated with EYLEA received 5 initial monthly doses in PHOTON and 3 in PULSAR.
In the first year, patients in the aflibercept 8 mg groups could have their dosing intervals shortened down to an every 8-week interval if protocol-defined criteria for disease progression were observed. Intervals could not be extended until the second year of the study, with those results still to be assessed.
Patients in all EYLEA groups maintained a fixed 8-week dosing regimen throughout their participation in the trials.
The lead sponsors of the trials were Regeneron for PHOTON and Bayer for PULSAR.