* Positive recommendation is based on two Phase III studies. In addition to

    robust retinal drying with Vabysmo, these data show early and sustained
    vision improvements, which are non-inferior to aflibercept
  * If approved, Vabysmo would be the first and only bispecific antibody
    treatment available for the nearly one million people with RVO in the
    European Union
  * Vabysmo is already approved in the US and Japan for RVO and in more than 95
    countries around the world for people living with nAMD and DME

Basel, 28 June 2024 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that
the European Medicines Agency's Committee for Medicinal Products for Human Use
(CHMP) has adopted a positive opinion for the extension of the Vabysmo®
(faricimab) marketing authorisation to include the treatment of visual
impairment due to macular edema secondary to retinal vein occlusion (RVO). A
final decision regarding the approval is expected from the European Commission
in the near future.
"This CHMP recommendation represents an important step towards bringing Vabysmo
to even more patients living with vision loss in Europe," said Levi Garraway,
M.D., Ph.D., Roche's Chief Medical Officer and Head of Global Product
Development. "Recognising the disruptive impact retinal vein occlusion can have
on the everyday lives and independence of these patients, we hope that Vabysmo
will offer a new treatment option that can effectively help preserve and improve
their vision."
The CHMP decision is based on full 72-week data from the Phase III BALATON and
COMINO studies evaluating Vabysmo in more than 1,200 people with macular edema
due to branch and central RVO (BRVO and CRVO).(1,2) In both studies, Vabysmo
demonstrated early and sustained vision improvements non-inferior to
aflibercept, and robust retinal drying. Vabysmo was well tolerated and the
safety profile was consistent with previous studies.(3 )Current available
treatments for RVO are typically given every one to two months.( 4)(,5)
Vabysmo was first approved for RVO by the United States Food and Drug
Administration in October 2023 and by the Japan Ministry of Health, Labour and
Welfare in March 2024.(6-8 )It is also approved in more than 95 countries around
the world for people living with neovascular or 'wet' age-related macular
degeneration (nAMD) and diabetic macular edema (DME).(6)(,8)(-11)
Roche has the broadest retina pipeline in ophthalmology. Led by science and
informed by insights from people with eye conditions, Roche is committed to
saving people's eyesight from the leading causes of vision loss through
pioneering treatments.
About retinal vein occlusion (RVO)
RVO is the second most common cause of vision loss due to retinal vascular
diseases. It affects an estimated 28 million adults globally, mainly those aged
60 or older, and can lead to severe and sudden vision loss.(12,13 )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 ischemia, 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 RVO, 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 RVO, which is less common, affecting more than four
million people worldwide, and occurs when the eye's central retinal vein becomes
blocked.(12,17)
About the BALATON and COMINO studies(1,2)
BALATON (NCT04740905) and COMINO (NCT04740931) were two randomised, multicentre,
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 six monthly injections of either Vabysmo (6.0 mg) or
aflibercept (2.0 mg). From weeks 24-72, 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 included change in central
subfield thickness and drying of retinal fluid from baseline over time up to
week 24.
About the Vabysmo® (faricimab) clinical development programme
Roche has a robust Phase III clinical development programme for Vabysmo. The
programme includes AVONELLE-X (NCT04777201), an extension study of TENAYA
(NCT03823287) and LUCERNE (NCT03823300), evaluating the long-term safety and
tolerability of Vabysmo in neovascular or 'wet' age-related macular degeneration
(nAMD), and RHONE-X (NCT04432831), an extension study of YOSEMITE (NCT03622580)
and RHINE (NCT03622593) 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 (NCT05224102) study of Vabysmo in
underrepresented patient populations with DME, the SALWEEN study of Vabysmo in a
subpopulation of nAMD highly prevalent in Asia, and the POYANG (NCT06176352)
study of Vabysmo in adult treatment-naive patients with choroidal
neovascularisation secondary to pathologic myopia.(20-22 )Roche has also
initiated the VOYAGER (NCT05476926) study, a global real-world data collection
platform, and supports several other independent studies to further understand
retinal conditions with a high unmet need.(23)
About Vabysmo® (faricimab)
Vabysmo is the first bispecific antibody approved for the eye. 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.(24) Vabysmo is approved in more
than 95 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 several
countries, including the US and Japan, for RVO.(4,6-9) 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 across multiple vision-threatening conditions,
including diabetic eye diseases, geographic atrophy and autoimmune conditions,
such as thyroid eye disease and uveitic macular edema.
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 was approved by the U.S. Food and Drug Administration in
2021.(25) Vabysmo is approved around the world for people living with
neovascular or 'wet' age-related macular degeneration and diabetic macular
edema, and in several countries, including the US and Japan for macular edema
following retinal vein occlusion.( 4)(,6)(-9) Lucentis® (ranibizumab injection)*
was the first treatment approved to improve vision in people with certain
retinal conditions.(5)
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial
manufacturers of branded medicines, Roche has grown into the world's largest
biotechnology company and the global leader in in-vitro diagnostics. The company
pursues scientific excellence to discover and develop medicines and diagnostics
for improving and saving the lives of people around the world. We are a pioneer
in personalised healthcare and want to further transform how healthcare is
delivered to have an even greater impact. To provide the best care for each
person we partner with many stakeholders and combine our strengths in
Diagnostics and Pharma with data insights from the clinical practice.
In recognising our endeavour to pursue a long-term perspective in all we do,
Roche has been named one of the most sustainable companies in the
pharmaceuticals industry by the Dow Jones Sustainability Indices for the
fifteenth consecutive year. This distinction also reflects our efforts to
improve access to healthcare together with local partners in every country we
work.
Genentech, in the United States, is a wholly owned member of the Roche Group.
Roche is the majority shareholder in Chugai Pharmaceutical, Japan.
For more information, please visit www.roche.com (http://www.roche.com).
*Lucentis® (ranibizumab injection) was developed by Genentech, a member of the
Roche Group. Genentech retains commercial rights in the United States and
Novartis has exclusive commercial rights for the rest of the world.
All trademarks used or mentioned in this release are protected by law.
References
(1) Clinical Trials.gov. A study to evaluate the efficacy and safety of
faricimab in participants with macular edema secondary to branch retinal vein
occlusion (RVO) (BALATON) (Internet; cited June 2024). Available from:
https://clinicaltrials.gov/ct2/show/NCT04740905.
(2) Clinical Trials.gov. A study to evaluate the efficacy and safety of
faricimab in participants with macular edema secondary to central retinal or
hemiretinal vein occlusion (COMINO) (Internet; cited June 2024). Available from:
https://clinicaltrials.gov/ct2/show/NCT04740931.
(3) Ghanchi, et al. Efficacy, safety, and durability of faricimab in macular
edema due to RVO: 72-week results from the Phase III BALATON and COMINO trials.
Poster presented at: ARVO Annual Meeting, May, 5-9 2024, Seattle WA, US. Poster
#A0388.
(4) U.S. Food and Drug Administration (FDA). Highlights of prescribing
information, aflibercept 2 mg. 2022. (Internet; cited June 2024). Available
from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/125387s076lbl.pd
f.
(5) FDA. Highlights of prescribing information, Lucentis. 2014. (Internet; cited
June 2024). Available from:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2012/125156s0069s0076lbl.pd
f.
(6) FDA. Highlights of prescribing information, Vabysmo. 2022. (Internet; cited
June 2024). Available from:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2023/761235s003lbl.pdf.
(7) FDA approves Genentech's Vabysmo for the treatment of RVO (Internet; cited
June 2024). Available from: https://www.gene.com/media/press-
releases/15009/2023-10-26/fda-approves-genentechs-vabysmo-for-the-treatment-of-
RVO.
(8) Chugai obtains regulatory approval for Vabysmo, the only bispecific antibody
in the ophthalmology field, for additional indication of macular edema
associated with RVO. (Internet; cited June 2024). Available from:
https://www.chugai-pharm.co.jp/english/news/detail/20240326160000_1054.html.
(9) European Medicines Agency. Summary of product characteristics, Vabysmo,
2022 (Internet; cited June 2024). Available
from: https://www.ema.europa.eu/en/documents/product-information/vabysmo-epar-
product-information_en.pdf.
(10) Chugai obtains regulatory approval for Vabysmo, the first bispecific
antibody in ophthalmology, for neovascular age-related macular degeneration
(nAMD) and diabetic macular edema (DME) (Internet; cited January 2024).
Available from: https://www.chugai-
pharm.co.jp/english/news/detail/20220328160002_909.html.
(11) Roche data on file.
(12) Song P, et al. Global epidemiology of RVO: a systematic review and meta-
analysis of prevalence, incidence and risk factors. J Glob Health.
2019;9:010427.
(13) Moorfields Eye Hospital, United Kingdom National Health Service Foundation
Trust. RVO (Internet; cited June 2024). Available from:
https://www.moorfields.nhs.uk/condition/retinal-vein-occlusion.
(14) Joussen et al. Angiopoietin/Tie2 signalling and its role in retinal and
choroidal vascular diseases: a review of preclinical data. Eye.
2021;35:1305-1316.
(15) Regula JT, et al. Targeting key angiogenic pathways with a bispecific
CrossMab optimised for neovascular eye diseases. EMBO Molecular Medicine.
2016;8:1265-88.
(16) Schmidt-Erfurth U, et al. Guidelines for the management of retinal vein
occlusion by the European society of retina specialists (EURETINA).
Ophthalmologica. 2019;242:123-162.
(17) Campochiaro P. Molecular pathogenesis of retinal and choroidal vascular
diseases. Prog Retin Eye Res. 2015;49:67-81.
(18) Clinical Trials.gov. A study to evaluate the long-term safety and
tolerability of Vabysmo in participants with nAMD (AVONELLE-X) (Internet; cited
June 2024). Available from: https://clinicaltrials.gov/ct2/show/NCT04777201.
(19) Clinical Trials.gov. A study to evaluate the long-term safety and
tolerability of Vabysmo in participants with DME (RHONE-X) (Internet; cited June
2024). Available from: https://clinicaltrials.gov/ct2/show/NCT04432831.
(20) Clinical Trials.gov. A study to investigate faricimab treatment response in
treatment-naïve, underrepresented patients with DME (ELEVATUM). (Internet; cited
June 2024). Available from: https://clinicaltrials.gov/ct2/show/NCT05224102.
(21) APVRS. Design and rationale of the SALWEEN Trial: A Phase IIIb/IV Study of
faricimab, a dual angiopoietin-2 and vascular endothelial growth factor-a
inhibitor, in patients with polypoidal choroidal vasculopathy. (Internet; cited
June 2024). Available from: https://2022.apvrs.org/abstract/?code=200351.
(22) A study to evaluate the efficacy and safety of faricimab in patients with
choroidal neovascularisation secondary to pathologic myopia (POYANG). (Internet;
cited June 2024). Available from:
https://classic.clinicaltrials.gov/ct2/show/NCT06176352.
(23) Clinical Trials.gov. A Real-World Study to Gain Clinical Insights Into
Roche Ophthalmology Products (VOYAGER). (Internet; cited January 2024).
Available from: https://clinicaltrials.gov/ct2/show/NCT05476926.
(24) Heier JS, et al. Efficacy, durability, and safety of intravitreal faricimab
up to every 16 weeks for nAMD (TENAYA and LUCERNE): two randomised, double-
masked, Phase III, non-inferiority trials. The Lancet. 2022;399:729-740.
(25) FDA. Highlights of prescribing information, Susvimo. 2021. (Internet; cited
June 2024). Available from:
https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/761197s000lbl.pdf.
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