- ELEVIDYS received conditional approval on May 13, 2025.
- Eligible patients are 3-7 years old, negative for anti-AAVrh74 antibodies.
- Two fatal liver failure cases reported in non-ambulatory patients.
- Roche Diagnostics launched Elecsys anti-AAVrh74 assay on February 1, 2026.
Product Launch
Chugai Pharmaceutical Co., Ltd. has launched ELEVIDYS, Japan's first regenerative medical product for Duchenne muscular dystrophy (DMD). The product targets ambulatory patients aged 3 to less than 8 years who are negative for anti-AAVrh74 antibodies.
Approval and Eligibility
ELEVIDYS received conditional and time-limited approval on May 13, 2025. Eligible patients must not have a deletion of exon 8 and/or exon 9 in the DMD gene.
Clinical Studies and Safety
The approval is based on global Phase III clinical study results, which showed clinically meaningful improvements in key secondary endpoints. However, two fatal cases of acute liver failure in non-ambulatory DMD patients treated with ELEVIDYS have been reported overseas. In response, safety measures have been reinforced, including revising the electronic package insert and educational materials.
Safety Measures
Chugai has established a framework for promoting appropriate use through industry-government-academia collaboration. Materials for healthcare professionals and patients have been developed, and a specialist consultation framework is in place. The BRIDGE-NMD expert panel will provide internet-based consultations following facility certification by the Japanese Society of Child Neurology.
Companion Diagnostic
Roche Diagnostics K.K. has launched the Elecsys anti-AAVrh74 assay in Japan as a companion diagnostic to determine eligibility for ELEVIDYS treatment. The assay was listed on the NHI reimbursement price list on February 1, 2026.
