Medii and Japanese Society of Child Neurology Jointly Build 'Physician Collaboration System' for Safety Management of Regenerative Medicine for Duchenne Muscular Dystrophy

Medii Co., Ltd. (Headquarters: Shinjuku-ku, Tokyo; CEO and Physician: Hiroki Yamada; hereinafter 'Medii'), in collaboration with the Japanese Society of Child Neurology, has established an expert consultation system to support the safety management and proper use of 'Elevidys® Intravenous Infusion' (hereinafter 'Elevidys'), the first regenerative medicine product for Duchenne Muscular Dystrophy (DMD) in Japan (Manufacturer and Distributor: Chugai Pharmaceutical Co., Ltd.). Full-scale operations involving actual case registrations began in early March 2026.

This initiative realizes case consultations to 'BRIDGE-NMD' (*1), an expert panel officially formed by the Japanese Society of Child Neurology, through 'Medii E-Consult,' a specialist consultation service for physicians operated by Medii. This framework is explicitly mentioned in the 'Proper Use Guide' for Elevidys and represents a new safety management structure for advanced medical care in Japan.

*1 BRIDGE-NMD (Building Recommendations in Developing Gene Therapy for Neuromuscular Disorders): An expert team composed of physicians specializing in pediatric neurology, pediatric hepatology, pediatric hepatic surgery, pediatric cardiology, pediatric immunology, pediatric nephrology, and pediatric hematology.

Background In fields like rare diseases where effective treatments are not yet sufficient, initiatives are underway to expedite the practical application of new drugs through conditional and time-limited approval systems. However, as these approvals are based on efficacy estimated from early data, verifying efficacy within a specified period is an indispensable condition for maintaining the approval.

Under these circumstances, on May 13, 2025, Chugai Pharmaceutical obtained manufacturing and sales approval for Elevidys as the first regenerative medicine product in Japan for DMD, a progressive intractable disease, falling under conditional and time-limited approval. Sales commenced on February 20, 2026. This product is a treatment designed to supplement the deficiency of dystrophin, the cause of DMD, before irreversible muscle damage occurs with disease progression, through a single administration.

In introducing treatment with this product, from the perspective of prioritizing patient safety and ensuring proper use, it is crucial for physicians from various specialized fields, including hepatology and cardiology, to make decisions on treatment eligibility and perform rapid and accurate management if side effects occur. Against this background, there was a need for a safety management system where expert specialists can promptly support the advanced clinical decisions of certified physicians (primary doctors) at prescribing facilities and accumulate appropriate management practices for individual cases.

Details of the Initiative This system realizes team-based medical care on a digital platform, where multiple experts support a single patient, centered on the primary doctor. It was jointly built by Medii and the Japanese Society of Child Neurology with the cooperation of Chugai Pharmaceutical.

Formation of a 'One Team' involving field physicians and experts across multiple departments In this system, the primary doctor and the BRIDGE-NMD expert panel participate in the same chat room to co-manage cases, share information, and conduct consultations. After the launch, there are plans to expand collaboration functions to include other hospital departments (such as hepatology and cardiology) and regional follow-up doctors, establishing a system to watch over a single patient from multiple perspectives. By seamlessly delivering expert knowledge to the field, it supports advanced clinical decisions by primary doctors and realizes a high-level safety management structure.

*This system does not perform diagnosis or treatment; it is a mechanism to support information sharing and consultation among physicians, predicated on clinical decisions by the primary doctor. *Medical advice within this framework is provided by the expert panel formed by the Japanese Society of Child Neurology. Medii and Chugai Pharmaceutical are not involved in clinical decisions for individual cases or the content of consultations.

Comments from Stakeholders Dr. Hirofumi Komaki, Director of the Translational Medical Center and Head of the Department of Child Neurology, National Center of Neurology and Psychiatry: 'How to deliver innovative therapeutic drugs safely and appropriately is an important mission for our society. Initially, we considered consultations via email, but there were limits in terms of the confidentiality of the clinical information to be shared and the accumulation of information. Medii E-Consult was able to clear these concerns and realize an ideal collaboration system in a secure environment. I believe this mechanism will become a model case for physician collaboration.'