Avastin Receives Approval for Additional Indication as the World’s First Treatment for Neurofibromatosis Type 2

• Provides a new treatment option as the world’s first therapeutic drug for neurofibromatosis type 2, a disease with limited treatment options
• Approval based on the results from an investigator-initiated Japanese Phase II clinical study

TOKYO, June 19, 2026 -- Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) today announced that it received regulatory approval from the Ministry of Health, Labour and Welfare (MHLW) for the additional indication of neurofibromatosis type 2 (NF2) for the anti-cancer agent/humanized anti-VEGF^*1 monoclonal antibody Avastin^® Intravenous Infusion 100 mg/4 mL and 400 mg/16 mL [generic name: bevacizumab (genetical recombination)] (hereinafter, “Avastin”). Avastin is the first drug approved in the world for the treatment of this disease.

“We are very pleased to deliver Avastin as the world’s first therapeutic drug for neurofibromatosis type 2 in Japan. NF2 is a rare disease that causes symptoms such as hearing loss and dizziness, significantly impacting patients’ daily lives, and there has been a strong need for effective treatment options. Avastin represents a new therapeutic option, suggesting potential for the maintenance or improvement of hearing and a trend toward tumor reduction. We will continue our efforts to promptly provide appropriate use information so that we can contribute to patient treatment and improvement in quality of life,” said Chugai’s President and CEO, Dr. Osamu Okuda.

This approval is based on the results from the investigator-initiated Japanese Phase II clinical study, the BeatNF2 study, which evaluated the efficacy and safety of Avastin in NF2.

Approval Information *Relevant sections only, with modifications underlined

Indications:
◯ Unresectable advanced or recurrent colorectal cancer
◯ Unresectable advanced or recurrent non-small cell lung cancer excluding squamous cell carcinoma
◯ Inoperable or recurrent breast cancer
◯ Malignant glioma
◯ Ovarian cancer
◯ Advanced or recurrent cervical cancer
◯ Unresectable hepatocellular carcinoma
◯ Neurofibromatosis type 2

Dosage and Administration:
<Neurofibromatosis type 2>
The usual adult dosage is 5 mg/kg (body weight) of bevacizumab (genetical recombination) administered by intravenous infusion every two weeks.

[Reference Information]

Chugai Files for Additional Indication of Avastin for the Treatment of Neurofibromatosis Type 2 (News release dated September 24, 2025)
https://www.chugai-pharm.co.jp/english/news/detail/20250924153000_1185.html

About the BeatNF2 study

The BeatNF2 study (FMU2019-01-NF2 study / jRCT2080224914) is an investigator-initiated, multicenter, domestic Phase II, placebo-controlled, double-blind, randomized clinical study conducted in Japan to evaluate the efficacy and safety of Avastin in patients with NF2, a rare hereditary disease. Twelve institutions in Japan, including Fukushima Medical University Hospital, participated in the study.
The study enrolled 62 patients. During the initial treatment period, Avastin or placebo was administered every two weeks through week 22. From week 24 to week 46, all patients received Avastin every two weeks. During the follow-up period, Avastin could be administered up to six times if the attending physician judged disease progression.
The primary endpoint, “the proportion of patients with improved hearing at 24 weeks after treatment initiation compared to baseline, based on the evaluation using maximum speech discrimination score^*2,” was 16.1% (5/31; 95% CI: 5.5-33.7) in the Avastin group and 3.2% (1/31; 95% CI: 0.1-16.7) in the placebo group, with no statistically significant difference (P = 0.0858). Meanwhile, improvement in hearing measures and a trend toward reduction in tumor volume, a secondary endpoint, were suggested during the treatment period. Regarding safety, adverse reactions were observed in 57.4% (35/61) of patients who received Avastin, and hypertension was the most common adverse reaction at 18.0% (11/61).

*1 VEGF：Vascular Endothelial Growth Factor
*2 Maximum speech discrimination score is an indicator of speech comprehension ability. It refers to the percentage of correct answers in a monosyllabic word recognition test at the volume level that yields the highest accuracy while adjusting sound intensity. The higher this value, the better one’s ability to accurately understand speech when sounds are audible. It also serves as a measure for evaluating the effectiveness of hearing aids.

About the Neurofibromatosis Type 2 (NF2)¹

NF2 is an autosomal dominant hereditary disease characterized by bilateral acoustic nerve tumors (vestibular schwannomas). Symptoms associated with these tumors include hearing loss, dizziness, unsteadiness, and tinnitus. Additionally, symptoms related to spinal schwannomas may include numbness, sensory impairment, and weakness in the limbs.
Management of vestibular schwannomas includes observation, surgery, and radiation therapy. While these tumors are benign and may show minimal growth, surgical removal may be performed when symptoms develop or tumor growth is evident, which may affect long-term prognosis. Preservation of hearing through surgery is difficult, and there remains a risk of postoperative neurological complications.
According to overseas reports, NF2 is a rare disease with an incidence of approximately 1 in 25,000 to 60,000 individuals. In Japan, approximately 800 patients submitted clinical registry data between 2009 and 2013. Onset is most common in individuals in their teens to twenties.

About Avastin

Avastin is an antibody medicine that binds specifically to VEGF², which plays an important role in angiogenesis essential for tumor growth and metastasis, thereby inhibiting its activity. In Japan, Avastin was launched in June 2007 and is positioned as one of the standard therapies in treatment guidelines for various cancers. It has been approved for seven indications: unresectable advanced or recurrent colorectal cancer, unresectable advanced or recurrent non-squamous non-small cell lung cancer, inoperable or recurrent breast cancer, malignant glioma, ovarian cancer, advanced or recurrent cervical cancer, and unresectable hepatocellular carcinoma.

Trademarks used or mentioned in this release are protected by law.

Sources:

1. Japan Intractable Diseases Information Center. Neurofibromatosis type II (designated intractable disease 34) [Internet; cited June 2026] Available from: https://www.nanbyou.or.jp/  (Japanese only)
2. Presta LG, Chen H, O’Connor SJ, Chisholm V, Meng YG, Krummen L, et al. Humanization of an anti-vascular endothelial growth factor monoclonal antibody for the therapy of solid tumors and other disorders. Cancer Res 1997;57:4593‑9. [Internet; cited June 2026] Available from: https://pubmed.ncbi.nlm.nih.gov/9377574/