Avastin® Shows Hearing And Disease Control Benefits In NF2: Chugai Submits MHLW Application

Chugai Pharmaceutical Co., Ltd., has submitted a regulatory application to Japan’s Ministry of Health, Labour and Welfare (MHLW) seeking approval for Avastin® (bevacizumab) to treat neurofibromatosis type 2 (NF2), a rare hereditary disorder. If approved, this would represent a new indication for the anti-VEGF monoclonal antibody, which is already widely used in oncology.

The filing is supported by results from the Phase II BeatNF2 study (jRCT2080224914), a Japanese, investigator-initiated, multicenter, randomized, double-blind, placebo-controlled trial. The study enrolled 62 patients with NF2 to evaluate whether Avastin improved hearing function, a key clinical challenge in this patient population. At 24 weeks, the primary endpoint assessed the proportion of patients showing improved hearing compared to baseline, using maximum speech discrimination scores.

Beyond the primary outcome, the trial also evaluated tumor volume, auditory function, NF2 severity score, and retreatment efficacy. After the initial 24-week blinded period, all patients received Avastin for up to 48 weeks. Results showed meaningful improvements in hearing and disease control, supporting the potential of Avastin as the first targeted treatment option for NF2 in Japan.