Sarepta Therapeutics Expands Access to Gene Therapy for Duchenne in Japan
Is the future of gene therapy for rare diseases finally within reach? Sarepta Therapeutics, Inc. a leader in precision genetic medicine for rare diseases, has announced the commercial launch of ELEVIDYS (delandistrogene moxeparvovec) in Japan. This launch, managed by Chugai Pharmaceutical Co., Ltd., follows the therapy’s inclusion in Japan’s National Health Insurance (NHI) price list, marking a significant milestone in expanding access to this groundbreaking treatment.
ELEVIDYS is the first gene therapy to be launched in Japan for Duchenne muscular dystrophy (DMD). The therapy is now available for ambulatory individuals with Duchenne ages 3 to less than 8 years old, who have a deletion of any portion or the entirety of exon 8 and/or exon 9 in the DMD gene, and who are negative for anti-AAVrh74 antibodies. “The commercial launch of ELEVIDYS in Japan marks an important step in expanding access to this therapy, where early intervention may offer the greatest opportunity to preserve muscle function,” said Louise Rodino-Klapac, Ph.D., president of research & development and technical operations, Sarepta.
Key Insights at a Glance
- Commercial Launch: ELEVIDYS is now commercially available in Japan, expanding access to gene therapy for Duchenne muscular dystrophy.
- Target Population: The therapy is available for ambulatory individuals with Duchenne ages 3 to less than 8 years old, with specific genetic deletions.
- Reimbursement: ELEVIDYS has been listed on Japan’s National Health Insurance (NHI) price list, ensuring coverage for eligible patients.
- Collaboration: Chugai Pharmaceutical Co., Ltd., a member of the Roche Group, will manage postmarketing clinical studies and surveillance in Japan.
Addressing the Urgency of Duchenne Muscular Dystrophy
Duchenne muscular dystrophy is a devastating genetic disorder that primarily affects boys, leading to progressive muscle degeneration and loss of mobility. The early intervention provided by ELEVIDYS is crucial, as it aims to preserve muscle function and slow disease progression. The approval of ELEVIDYS in Japan is based on robust clinical data, including results from the global Phase 3 EMBARK study, which demonstrated clinically meaningful improvements in key motor function measures. Longer-term follow-up data have shown sustained functional efficacy and slower disease progression, with no new treatment-related safety signals observed in ambulatory patients. This underscores the critical need for timely access to advanced therapies like ELEVIDYS to improve the quality of life for those affected by Duchenne.
Why the Window for Action Is Closing Fast
Just as a ticking clock signals the urgency of a deadline, the rapid progression of Duchenne muscular dystrophy highlights the critical need for early intervention. The commercial launch of ELEVIDYS in Japan is a significant step in ensuring that eligible patients receive this potentially life-changing therapy. Chugai Pharmaceutical Co., Ltd., will be responsible for postmarketing clinical studies and all case postmarketing surveillance in Japan, further solidifying the commitment to long-term efficacy and safety. The first commercial sale of ELEVIDYS in Japan will also trigger a $40 million milestone payment to Sarepta, underscoring the financial and therapeutic significance of this launch.
Sarepta Therapeutics Mobilizes for Duchenne Muscular Dystrophy
Sarepta Therapeutics is committed to advancing rigorous science and generating long-term clinical and real-world evidence to inform care for the Duchenne community worldwide. The company’s collaboration with Roche, through which Chugai Pharmaceutical Co., Ltd., manages commercialization in Japan, is a testament to the global effort to transform the future for those living with Duchenne. Sarepta Therapeutics is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing. The company’s dedication to expanding access to this therapy and its ongoing research and development efforts are pivotal in the fight against Duchenne muscular dystrophy. Moving forward, Sarepta Therapeutics will continue to focus on advancing the science and ensuring that ELEVIDYS reaches as many eligible patients as possible.
Future Outlook
The launch of ELEVIDYS in Japan is like the opening of a new chapter in the book of Duchenne muscular dystrophy treatment. It represents a significant step forward in providing hope and potential relief to families affected by this devastating disease. With over 1,200 patients globally already benefiting from ELEVIDYS, the future looks promising for the Duchenne community. The three-year results from the EMBARK study, to be shared at an upcoming medical congress, will provide further insights into the long-term efficacy and safety of the therapy.
Conclusion
The commercial launch of ELEVIDYS in Japan by Sarepta Therapeutics and Chugai Pharmaceutical Co., Ltd., marks a pivotal moment in the treatment of Duchenne muscular dystrophy. By expanding access to this innovative gene therapy, the companies are taking a significant step toward improving the lives of affected individuals and their families. Join the conversation in the comments below.
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