Givinostat Receives Orphan Drug Designation for Duchenne Muscular Dystrophy in Japan – JCR Pharmaceuticals

JCR Pharmaceuticals Co., Ltd. announced that the Ministry of Health, Labour and Welfare of Japan granted orphan drug designation to givinostat, an investigational drug for the treatment of Duchenne muscular dystrophy (DMD).

Givinostat (marketed as Duvyzat in the US, EU, and the UK) is an orally administered histone deacetylase (HDAC) inhibitor developed by Italfarmaco S.p.A. It has been approved in multiple countries and regions, including the US, EU, and the UK, and is prescribed to patients aged 6 years and older with DMD in accordance with local prescribing information. It is currently not approved in Japan.

In December 2025, JCR entered into an exclusive license agreement with Italfarmaco for the development and commercialization of givinostat in Japan. JCR is currently advancing development toward obtaining marketing approval in Japan and plans to submit a marketing authorization application for the product in Japan by the end of 2026. In addition to receiving orphan drug designation in Japan, givinostat has also been granted Priority Examination status.

“The designation of givinostat as both an orphan drug and a product eligible for Priority Examination represents an important milestone toward its commercialization in Japan.” – Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals.

“We remain committed to bringing this new treatment option to patients with DMD in Japan as quickly as possible, addressing the significant unmet medical needs of the DMD community.” – Hiroyuki Sonoda, Ph.D., President and Chief Scientific Officer of JCR Pharmaceuticals.

JCR remains committed to advancing research and development to help meet the expectations of patients and families waiting for effective treatment options.