Genentech, a member of the Roche Group announced that the U.S. Food and Drug Administration (FDA) has accepted the company’s supplemental Biologics License Application (sBLA) for Vabysmo (faricimab-svoa) for the treatment of macular edema following retinal vein occlusion (RVO).
The sBLA is based on results from the phase 3 BALATON and COMINO studies that demonstrated treatment with Vabysmo provided early and sustained improvement in vision, meeting the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to aflibercept. Vabysmo’s safety profile was consistent with previous trials.
“This acceptance brings us one step closer to delivering Vabysmo as a treatment for retinal vein occlusion, a disease that affects more than one million people in the United States and can cause severe and sudden vision loss,” said Levi Garraway, MD, PhD, CMO, and head of Global Product Development. “If approved, this would be the third indication for Vabysmo, the first bispecific antibody available for the treatment of retinal conditions that can cause blindness.”
The data from the BALATON and COMINO studies will be submitted to other health authorities around the world, including the European Medicines Agency, for approval for the treatment of macular edema following RVO. The studies are ongoing, and data from weeks 24 to 72 will assess the potential of Vabysmo to extend dosing intervals up to every four months.
Vabysmo is the first bispecific antibody approved for the eye and was approved in the United States for the treatment of wet, or neovascular, age-related macular degeneration (AMD) and diabetic macular edema (DME) in January 2022. Vabysmo is approved in 60 countries, including Japan, the United Kingdom, and in the European Union, for wet AMD and DME.
Wet AMD, DME, and RVO together affect around three million people in the United States and are among the leading causes of vision loss.
Vabysmo’s efficacy and safety profile in wet AMD and DME is supported by four large, global studies involving more than 3,000 participants and extensive real world experience, with nearly one million doses distributed globally.
Vabysmo is the only injectable eye medicine approved for wet AMD and DME by the FDA with the option for treatments from one to four months apart in the first year following four initial monthly loading doses, based on evaluation of the patient’s anatomy and vision outcomes. It targets and inhibits two disease pathways linked to a number of vision-threatening retinal conditions by neutralizing angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A).
About the BALATON and COMINO studies
BALATON (NCT04740905) and COMINO (NCT04740931) are two randomized, multicenter, double-masked, global Phase III studies evaluating the efficacy and safety of Vabysmo®️ (faricimab-svoa) compared to aflibercept. For the first 20 weeks, patients are randomized 1:1 to receive six monthly injections of either Vabysmo (6.0 mg) or aflibercept (2.0 mg). From weeks 24 to 72, all patients receive Vabysmo (6.0 mg) up to every four months – according to a personalized treatment interval dosing regimen – using a treat-and-extend approach.
The BALATON study is being conducted in 553 patients with branch retinal vein occlusion. The COMINO study is being conducted in 729 patients with central retinal or hemiretinal vein occlusion.
The primary endpoint of each study is the change in best-corrected visual acuity (BCVA) from baseline at 24 weeks. Secondary endpoints include change in central subfield thickness (CST) from baseline over time up to 24 weeks.
Both studies met their primary endpoint, with Vabysmo showing non-inferior visual acuity gains compared to aflibercept. The average vision gains from baseline were comparable between the two treatments in both studies. In BALATON, vision gains were +16.9 eye chart letters in the Vabysmo arm and +17.5 letters in the aflibercept arm at 24 weeks. In COMINO, vision gains were +16.9 letters in the Vabysmo arm and +17.3 letters in the aflibercept arm at 24 weeks.
A secondary endpoint showed that Vabysmo achieved rapid and robust drying of retinal fluid, as measured by reduction in CST from baseline. In both studies, reductions in CST were comparable across treatment arms. In BALATON, CST reductions from baseline at 24 weeks were 311.4 μm in the Vabysmo arm and 304.4 μm in the aflibercept arm. In COMINO, CST reductions from baseline at 24 weeks were 461.6 μm in the Vabysmo arm and 448.8 μm in the aflibercept arm.
Additionally, both studies showed that more Vabysmo patients had an absence of blood vessel leakage in the retina compared to aflibercept patients as seen in a pre-specified exploratory endpoint. Blood vessel leakage in the macula may lead to more retinal fluid, which can cause swelling and blurry vision.
In BALATON, one-third of patients (34%) treated with Vabysmo had an absence of macular leakage compared to one-fifth (21%) of aflibercept patients at 24 weeks. In COMINO, the rates were 44% for Vabysmo patients versus 30% for aflibercept patients at 24 weeks.
In both studies, Vabysmo’s safety profile was consistent with previous trials. The most common adverse reaction was conjunctival hemorrhage (3%). Safety results were consistent across study arms.
The studies are ongoing, and data from weeks 24 to 72 will assess the potential of Vabysmo to extend dosing intervals up to every four months.