RVO is the third indication for Vabysmo, in addition to neovascular or ‘wet’ age-related macular degeneration and diabetic macular edema
Approval is based on two phase III studies demonstrating early and sustained vision improvements that were non-inferior to aflibercept
Vabysmo also demonstrated rapid and robust drying of retinal fluid
Additional U.S. label update across indications includes information on rare post-marketing reports of retinal vasculitis and/or retinal vascular occlusion; reporting rate is in line with other broadly used intravitreal treatments
Basel, 27 October 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the United States Food and Drug Administration (U.S. FDA) has approved Vabysmo® (faricimab) for the treatment of macular edema following retinal vein occlusion (RVO). RVO is the third indication for Vabysmo, in addition to neovascular or ‘wet’ age-related macular degeneration (nAMD) and diabetic macular edema (DME).1 Together, the three retinal conditions affect around 70 million people worldwide and are among the leading causes of vision loss.2-5
“Vabysmo is a new treatment option for RVO that can help people preserve and improve their vision, with the added benefit of retinal drying,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “The efficacy and safety profile of Vabysmo has been well established in global clinical trials and is reinforced by a growing breadth of real-world evidence, with hundreds of thousands of people treated.”
Vabysmo is the first and only bispecific antibody approved for the eye.1,6 Today’s approval in RVO is based on positive results from the global phase III BALATON and COMINO studies that demonstrated monthly treatment with Vabysmo provided early and sustained improvement in vision in people with branch and central RVO, meeting the primary endpoint of non-inferior visual acuity gains at 24 weeks compared to aflibercept. This was further supported by data showing Vabysmo achieved rapid and robust drying of retinal fluid. In BALATON and COMINO, Vabysmo was generally well tolerated and the safety profile was consistent with previous trials. The most common adverse reaction was conjunctival haemorrhage (3%). Safety results were consistent across study arms.7-9
Information has also been added to the Warnings and Precautions section of the U.S. label based on rare post-marketing cases of retinal vasculitis and/or retinal vascular occlusion, typically in the presence of intraocular inflammation. The reported rate of retinal vasculitis with vascular occlusion is 0.06 per 10,000 injections, in line with real-world reported frequencies of other broadly used intravitreal treatments for people living with nAMD, DME and RVO.10-12
To date, Vabysmo is approved in more than 80 countries around the world for people living with nAMD and DME, with approximately 2 million doses distributed globally
About retinal vein occlusion (RVO)
RVO is the second most common cause of vision loss due to retinal vascular conditions. It affects an estimated 28 million adults globally, mainly those aged 60 or older, and can lead to severe and sudden vision loss.2,13 The level of angiopoietin-2 (Ang-2) is elevated in RVO and it is thought that increased Ang-2 expression drives disease progression.14,15 RVO typically results in sudden, painless vision loss in the affected eye because the vein blockage restricts normal blood flow in the affected retina, resulting in ischaemia, bleeding, fluid leakage and retinal swelling called macular edema.13,16,17 Currently, macular edema due to RVO is typically treated with repeated intravitreal injections of anti-vascular endothelial growth factor therapies.16 There are two main types of RVO: branch retinal vein occlusion, which affects more than 23 million people globally and occurs when one of the four smaller ‘branches’ of the main central retinal vein becomes blocked; and central retinal vein occlusion, which is less common, affecting more than four million people worldwide, and occurs when the eye’s central retinal vein becomes blocked.
About the BALATON and COMINO studies
BALATON (NCT04740905) and COMINO (NCT04740931) are two randomised, multicentre, double-masked, global phase III studies evaluating the efficacy and safety of Vabysmo®️ (faricimab) compared to aflibercept. For the first 20 weeks, patients were randomised 1:1 to receive monthly injections for six months of either Vabysmo (6.0 mg) or aflibercept (2.0 mg). From weeks 24 to 72, all patients received Vabysmo (6.0 mg) up to every four months, using a treat-and-extend dosing regimen.
The BALATON study was conducted in 553 people with branch retinal vein occlusion. The COMINO study was conducted in 729 people with central retinal or hemiretinal vein occlusion. The primary endpoint of each study was the change in best-corrected visual acuity from baseline at 24 weeks. Secondary endpoints (weeks 0-24) included change in central subfield thickness and drying of retinal fluid, from baseline over time up to week 24. Secondary endpoints (weeks 24-72) were treatment durability at 68 weeks and continuation of weeks 0-24 endpoints.
About the Vabysmo® (faricimab) clinical development programme
Roche has a robust phase III clinical development programme for Vabysmo. The programme includes AVONELLE-X, an extension study of TENAYA and LUCERNE, evaluating the long-term safety and tolerability of Vabysmo in neovascular or ‘wet’ age-related macular degeneration (nAMD), and RHONE-X, an extension study of YOSEMITE and RHINE, evaluating the long-term safety and tolerability of Vabysmo in diabetic macular edema (DME).18,19 Roche has also initiated several phase IV studies, including the ELEVATUM study of Vabysmo in underrepresented patient populations with DME, the SALWEEN study of Vabysmo in a subpopulation of nAMD highly prevalent in Asia, as well as the VOYAGER study, a global real-world data collection platform.20-22 Roche also supports several other independent studies to further understand retinal conditions with a high unmet need.
About Vabysmo® (faricimab)
Vabysmo is the first bispecific antibody approved for the eye.1,6 It targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions by neutralising angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A). Ang-2 and VEGF-A contribute to vision loss by destabilising blood vessels, causing new leaky blood vessels to form and increasing inflammation. By blocking pathways involving Ang-2 and VEGF-A, Vabysmo is designed to stabilise blood vessels.23,24 Vabysmo is approved in more than 80 countries around the world, including the United States, Japan, the United Kingdom and the European Union for people living with neovascular or ‘wet’ age-related macular degeneration and diabetic macular edema, and in the United States for people living with macular edema following retinal vein occlusion. Review by other regulatory authorities is ongoing
About Roche in ophthalmology
Roche is focused on saving people’s eyesight from the leading causes of vision loss through pioneering therapies. Through our innovation in the scientific discovery of new potential drug targets, personalised healthcare, molecular engineering, biomarkers and continuous drug delivery, we strive to design the right therapies for the right patients.
We have the broadest retina pipeline in ophthalmology, which is led by science and informed by insights from people with eye diseases. Our pipeline includes gene therapies and treatments for geographic atrophy and other vision-threatening diseases, including rare and inherited conditions.
Applying our extensive experience, we have already brought breakthrough ophthalmic treatments to people living with vision loss. Susvimo™ (previously called Port Delivery System with ranibizumab) 100 mg/mL for intravitreal use via ocular implant is the first United States Food and Drug Administration-approved refillable eye implant for neovascular or ‘wet’ age-related macular degeneration that continuously delivers a customised formulation of ranibizumab over a period of months.27 Vabysmo® (faricimab) is the first bispecific antibody approved for the eye, which targets and inhibits two signalling pathways linked to a number of vision-threatening retinal conditions.1,6,23,24 Lucentis® (ranibizumab injection)^ is the first treatment approved to improve vision in people with certain retinal conditions.
About Roche
Roche is a global pioneer in pharmaceuticals and diagnostics focused on advancing science to improve people’s lives. The combined strengths of pharmaceuticals and diagnostics under one roof have made Roche the leader in personalised healthcare – a strategy that aims to fit the right treatment to each patient in the best way possible.
Roche is the world’s largest biotech company, with truly differentiated medicines in oncology, immunology, infectious diseases, ophthalmology and diseases of the central nervous system. Roche is also the world leader in in vitro diagnostics and tissue-based cancer diagnostics, and a frontrunner in diabetes management.
Founded in 1896, Roche continues to search for better ways to prevent, diagnose and treat diseases and make a sustainable contribution to society. The company also aims to improve patient access to medical innovations by working with all relevant stakeholders. More than thirty medicines developed by Roche are included in the World Health Organization Model Lists of Essential Medicines, among them life-saving antibiotics, antimalarials and cancer medicines. Moreover, for the twelfth consecutive year, Roche has been recognised as one of the most sustainable companies in the Pharmaceuticals Industry by the Dow Jones Sustainability Indices (DJSI).
The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan