Chugai wins MHLW approval for Avastin as first treatment for neurofibromatosis type 2
- MHLW approved bevacizumab (Avastin) for neurofibromatosis type 2, the first therapeutic drug worldwide for this indication.
- The approval was based on the investigator-initiated BeatNF2 Phase II randomized, double-blind, placebo-controlled study in Japan that enrolled 62 patients across 12 institutions.
- Primary endpoint (hearing improvement at 24 weeks by maximum speech discrimination score): Avastin 16.1% (5/31) vs placebo 3.2% (1/31), P = 0.0858; secondary endpoints suggested hearing improvements and a trend toward tumor-volume reduction.
- Recommended adult dosage for NF2 is 5 mg/kg IV every two weeks; adverse reactions occurred in 57.4% of Avastin recipients, with hypertension the most common (18.0%).
Approval
The Ministry of Health, Labour and Welfare granted an additional indication for bevacizumab (Avastin) for neurofibromatosis type 2 (NF2), making it the first drug approved worldwide for this disease.
BeatNF2 study
The decision was based on the investigator-initiated BeatNF2 study (jRCT2080224914), a multicenter, randomized, double-blind, placebo-controlled Phase II trial in Japan that enrolled 62 patients at 12 institutions. Patients received Avastin or placebo every two weeks through week 22; from week 24 to 46 all patients received Avastin, and up to six additional doses were allowed in follow-up if progression was judged by the treating physician.
Efficacy
The primary endpoint—proportion of patients with improved hearing at 24 weeks by maximum speech discrimination score—was 16.1% (5/31; 95% CI 5.5–33.7) with Avastin versus 3.2% (1/31; 95% CI 0.1–16.7) with placebo (P = 0.0858). Secondary analyses indicated improvements in hearing measures and a trend toward reduction in tumor volume during treatment.
Dosage and safety
The prescribed adult dose for NF2 is 5 mg/kg administered by intravenous infusion every two weeks. Adverse reactions were reported in 57.4% (35/61) of patients receiving Avastin, with hypertension the most frequent adverse reaction at 18.0% (11/61).
Source: Chugai Pharmaceutical
