Sarepta Therapeutics has now officially announced the launch of Sarepta ELEVIDYS in Japan. Chugai Pharmaceutical Co., Ltd. is now in charge of selling the drug all over the country. Japan’s National Health Insurance recently added the therapy to the list of things it will pay for. This rollout guarantees Sarepta a $40 million milestone payment. Also, this is the first DMD gene therapy that can be bought in Japan.
The therapy is for kids aged three to seven who can walk and have certain genetic mutations. Before treatment, patients must also test negative for anti-AAVrh74 antibodies. Early intervention is still very important for keeping young boys’ muscles working. So, last May, the Ministry of Health, Labour, and Welfare gave its approval.
“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. “We are delighted that ELEVIDYS is now available to eligible patients in Japan and remain focused on advancing rigorous science and generating long-term clinical and real-world evidence to help inform care for the Duchenne community worldwide.”
Working together and growing around the world
Approval came after the global Phase 3 EMBARK study showed good results. This trial demonstrated significant motor enhancements in ambulatory patients. Data also shows that the disease is progressing more slowly than in other groups. Chugai is now in charge of all postmarketing surveillance for the Roche Group.
Sarepta and Roche are working together to change the future for Duchenne patients. Sarepta makes the drugs in the U.S., and Roche gets the drugs approved for sale in other countries. More than 1,200 patients around the world have already gotten the one-dose treatment. This partnership keeps using cutting-edge genetic medicine to protect muscle function. The treatment sends a transgene to skeletal muscles to make micro-dystrophin. It specifically goes after the genetic cause of the disease. As a result, this sets a new standard of care for families in Japan.
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News Source: Businesswire.com
