JCR Pharmaceuticals (TSE: 4552) has decided how it will try to bring a Duchenne muscular dystrophy drug to Japanese patients: file on the strength of trials already run abroad rather than wait on a full domestic pivotal study. Following a face-to-face consultation with the Pharmaceuticals and Medical Devices Agency, the company said it will submit an application for manufacturing and marketing approval of givinostat, sold overseas as Duvyzat, before the end of 2026, using foreign clinical trial results as the primary evidence. JCR is targeting approval and a Japan launch within 2027.
| Milestone | Target |
|---|---|
| PMDA face-to-face consultation | Completed; result shaped the filing plan |
| Marketing approval application | To be filed in Japan by the end of 2026, based mainly on overseas trial data |
| Approval and Japan launch | Targeted within 2027 |
| Japan-specific pharmacokinetics/safety trial | Separate track, independent of the approval application |
Alongside the filing, JCR Pharma will run a separate trial in Japan to check the drug's pharmacokinetics and safety in Japanese DMD patients. The company frames this study as independent of the approval application itself, a structure meant to let the marketing filing move forward without waiting on local trial enrollment.
Givinostat is an oral histone deacetylase inhibitor that JCR licensed from Italy's Italfarmaco S.p.A.; the company announced the Japanese development and commercialization rights in December. The drug is already approved for DMD patients aged six and over in the United States, the European Union and the United Kingdom. JCR estimates roughly 3,500 people in Japan have the disease, which is caused by mutations in the dystrophin gene. JCR says the drug's mechanism, which targets HDAC activity rather than any single dystrophin mutation, means it can be used regardless of a patient's specific genetic subtype. The company called the financial impact of the plan on the current fiscal year ending March 2027 negligible.