Japan’s MHLW approves Avastin® (bevacizumab) as the first-ever therapy for Neurofibromatosis Type 2, offering patients a non-surgical option to preserve hearing and slow tumor progression.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
Chugai Pharmaceutical Co., Ltd. announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has approved an additional indication for Avastin® (bevacizumab) for the treatment of Neurofibromatosis Type 2 (NF2). The decision makes Avastin the first medicine approved specifically for NF2-associated vestibular schwannomas anywhere in the world and expands its use beyond seven previously approved oncology indications in Japan.
About NF2
NF2 is a rare autosomal dominant genetic disorder characterized by the development of bilateral vestibular schwannomas, benign tumors that arise on the hearing and balance nerves. The disease typically develops during adolescence or early adulthood and can lead to progressive hearing loss, tinnitus, dizziness, balance impairment, and other neurological complications. Treatment options have historically been limited to observation, surgery, and radiation therapy.
Under the newly approved indication, Avastin is administered intravenously at a dose of 5 mg/kg every two weeks in adult patients with NF2.
Approval Supported by BeatNF2 Study
The approval was supported by data from the BeatNF2 study (FMU2019-01-NF2; jRCT2080224914), an investigator-initiated, multicenter, randomized, double-blind, placebo-controlled Phase II trial conducted at 12 institutions across Japan.
The study enrolled 62 patients with NF2 and evaluated the efficacy and safety of Avastin. The primary endpoint assessed the proportion of patients achieving hearing improvement at 24 weeks based on the maximum speech discrimination score.
Hearing improvement was observed in 16.1% of patients treated with Avastin compared with 3.2% of patients receiving placebo. Although the difference did not reach statistical significance (P=0.0858), secondary analyses demonstrated improvements in hearing-related outcomes and a favorable trend toward reduction in vestibular schwannoma volume. These findings contributed to the overall assessment of clinical benefit supporting regulatory approval.
Safety Profile
The safety profile observed in the study was consistent with the established safety profile of bevacizumab. Adverse reactions were reported in 57.4% of patients receiving Avastin, with hypertension being the most common adverse event, occurring in 18.0% of patients. No new safety signals were identified.
Clinical Implications
Commenting on the approval, Dr. Osamu Okuda, President and Chief Executive Officer of Chugai Pharmaceutical, said, “We are very pleased to deliver Avastin as the world’s first therapeutic drug for neurofibromatosis type 2 in Japan. 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 provide appropriate use information to support patient treatment and quality of life.”
What This Means for Patients
For people living with Neurofibromatosis Type 2, this approval brings the first medicine specifically approved for their condition. Until now, many patients have relied on regular monitoring, surgery, or radiation treatment to manage the disease. Avastin offers a new treatment option that may help preserve hearing and delay worsening symptoms for some patients. While it may not work for everyone, the availability of an approved therapy provides new hope for individuals and families affected by Neurofibromatosis Type 2, a condition that can have a major impact on hearing, balance, independence, and overall quality of life.
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About the Writer
Chikkula Pavan Kumar (LinkedIn), PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.