Chugai Pharmaceutical Co., Ltd. announced on May 19, 2025, that it has received regulatory approval from Japan's Ministry of Health, Labour and Welfare (MHLW) for Vabysmo (faricimab) to treat choroidal neovascularization associated with angioid streaks. This marks a significant milestone as Vabysmo becomes the first approved treatment for this rare eye condition in Japan.
The approval follows positive results from the Phase III NIHONBASHI study, which were presented at the 129th Annual Meeting of the Japanese Ophthalmological Society on April 17. The study demonstrated that Vabysmo effectively improved vision in Japanese patients with angioid streaks, a disease characterized by cracks in the retina that can lead to vision impairment.
"We are very pleased that we can offer Vabysmo as the first approved treatment in Japan for choroidal neovascularization associated with angioid streaks," said Dr. Osamu Okuda, Chugai's President and CEO. "This disease is known to cause vision impairment and has a poor prognosis. We expect that treatment with Vabysmo will contribute to maintaining and improving vision for patients."
Clinical Trial Results
The NIHONBASHI study was a multicenter, open-label, single-arm Phase III trial conducted in Japan. The study enrolled 24 patients with angioid streaks who received Vabysmo every four weeks for the first three doses, followed by treatment as needed based on disease activity assessments at 4-week intervals.
The primary endpoint of mean change in best-corrected visual acuity from baseline at week 12 was +5.8 letters (90% confidence interval: 3.0 to 8.5 letters). The lower limit of the confidence interval exceeded the pre-specified clinically meaningful threshold of 0 letters, demonstrating statistically significant visual acuity improvement.
Additionally, the study showed a mean reduction in central retinal thickness of 106.4 μm (90% confidence interval: -123.4 to -89.4) from baseline at week 12, indicating a decrease in retinal fluid that can cause retinal edema and blurred vision.
Safety Profile
Vabysmo was generally well-tolerated during the trial, with a safety profile consistent with previous studies. Adverse events in the study eye occurred in 20.8% (5/24) of patients, with no serious ocular adverse events or events leading to study drug discontinuation. Non-ocular adverse events occurred in 50.0% (12/24) of patients. Serious adverse events were reported in two patients, but none were assessed as related to Vabysmo treatment.
About Angioid Streaks
Angioid streaks is a rare condition characterized by cracks in parts of the retina, causing discoloration (pigmented streaks) of the fundus. While often asymptomatic initially, when choroidal neovascularization extends to the macula region, it causes symptoms such as decreased or distorted vision.
The exact prevalence of angioid streaks in Japan is unknown, but it's estimated that approximately 300 patients have pseudoxanthoma elasticum, a designated intractable disease associated with angioid streaks in approximately half of cases.
Conventional treatments such as surgery and laser therapy have not proven sufficiently effective for this condition, highlighting the significant unmet medical need that Vabysmo now addresses.
Innovative Mechanism of Action
Vabysmo is the first bispecific antibody approved for ophthalmic use. It works by simultaneously targeting and inhibiting two signaling pathways linked to vision-threatening retinal conditions: angiopoietin-2 (Ang-2) and vascular endothelial growth factor-A (VEGF-A).
Both Ang-2 and VEGF-A contribute to vision loss by destabilizing blood vessels, causing new leaky blood vessels to form, and increasing inflammation. By blocking both pathways, Vabysmo is designed to stabilize blood vessels and potentially provide more comprehensive disease control than single-target therapies.
Regulatory Status
Vabysmo is now approved in nearly 100 countries worldwide for various retinal conditions, including neovascular age-related macular degeneration, diabetic macular edema, and macular edema following retinal vein occlusion. The new indication for angioid streaks in Japan represents an important expansion of its therapeutic applications.
The drug was granted priority review status by Japanese regulators, reflecting the significant unmet need for effective treatments for angioid streaks. Chugai has committed to providing comprehensive information on the proper use of Vabysmo to healthcare professionals to ensure optimal patient outcomes.
"Based on the study results, Vabysmo is expected to become the first drug for angioid streaks, for which there are currently no approved drugs in Japan," Dr. Okuda had stated following the presentation of the NIHONBASHI study results. "Chugai will make every effort to deliver this drug to patients who are eagerly awaiting treatment as soon as possible."
With this approval, Japanese patients with angioid streaks now have access to a targeted therapy that has demonstrated efficacy in improving visual outcomes for this challenging condition.
