The Japanese Ministry of Health, Labour, and Welfare (MHLW) has granted approval to Sarepta Therapeutics’ gene therapy Elevidys (delandistrogene moxeparvovec) for treating Duchenne muscular dystrophy (DMD) in patients aged between three to eight years old.
This marks the first global approval for DMD treatment in patients younger than four years old.
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The approval is intended for individuals with deletions in exon 8 and/or exon 9 of the DMD gene, and are negative for anti-adeno-associated virus rhesus isolate serotype 74 (AAVrh74) antibodies.
MHLW’s decision is conditional and time-limited, allowing marketing authorisation in the country for up to seven years for medicines addressing serious conditions.
The approval in Japan was based on the safety and efficacy data for the therapy, including muscle health and longer-term functional outcomes from the Elevidys clinical programmes, alongside two-year outcomes from the global Phase III EMBARK trial.
According to Sarepta, the long-term functional results demonstrated significant improvements in several motor function measures for Elevidys-treated patients against a matched external control group, without any safety concerns noted over the two-year trial period.
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By GlobalDataWith this approval, the company is set to receive up to $103.5m in milestone payments related to regulatory and commercial achievements.
Sarepta Therapeutics’ research and development head and chief scientific officer Louise Rodino-Klapac said: “With the approval of Elevidys in Japan, children who are living with this rare, fatal disease, one marked by progressive muscle deterioration and weakness, now have an additional treatment option.
“Sarepta is committed to supporting our partners so we can bring this treatment to additional people with Duchenne around the world who urgently need a therapy that potentially addresses the root cause of the disease.”
Under a 2019 partnership agreement, Sarepta and Roche are working together to bring Elevidys to the Duchenne community.
Elevidys is a single-dose AAV-based gene transfer therapy that aims to address the genetic root of DMD.
Last November, Arrowhead Pharmaceuticals entered a global licensing and partnership deal with Sarepta to develop rare genetic disease treatments.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
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