HYOGO, Japan — September 28, 2026
JCR Pharmaceuticals Co., Ltd. announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug designation to givinostat, an investigational treatment for Duchenne muscular dystrophy (DMD). Givinostat, marketed as Duvyzat in the United States, European Union and United Kingdom, is an orally administered histone deacetylase (HDAC) inhibitor developed by Italfarmaco and currently approved in multiple countries for patients aged 6 years and older with DMD. The therapy is not currently approved in Japan. JCR entered an exclusive license agreement with Italfarmaco in December 2025 to develop and commercialize givinostat in Japan and plans to submit a Japanese marketing authorization application by the end of 2026. The product has also received Priority Examination status in Japan.
Givinostat Receives Dual Regulatory Development Designations
The orphan drug designation and Priority Examination status provide regulatory development support as JCR prepares givinostat for potential commercialization in Japan. Japan’s orphan drug framework provides special measures to support development of medicines for serious rare diseases with significant unmet medical needs. Eligible products generally target conditions affecting fewer than 50,000 people in Japan or designated intractable diseases, while applicants must demonstrate an appropriate development plan and resources. JCR said the designations represent an important milestone in its Japanese development strategy for givinostat. The company is advancing the product toward a marketing authorization application planned for submission by the end of 2026. Any potential approval would remain subject to the MHLW’s regulatory review of the application and supporting clinical and quality data.
Oral HDAC Inhibitor Targets DMD Biology
Givinostat is designed to inhibit histone deacetylase activity associated with Duchenne muscular dystrophy muscle, with the goal of regulating biological processes involved in muscle maintenance and repair. According to JCR, the mechanism is independent of the specific dystrophin gene mutation responsible for DMD. DMD is a progressive neuromuscular disorder caused by mutations in the dystrophin gene, resulting in the absence or dysfunction of functional dystrophin and increasing vulnerability of muscle fibers to damage. Progressive muscle degeneration can lead to worsening weakness, loss of ambulation and involvement of cardiac and respiratory muscles. JCR estimates that approximately 3,500 people in Japan live with DMD. Givinostat is currently approved in the United States, European Union and United Kingdom, but its development and regulatory status in Japan remains separate and requires Japanese authorization.
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JCR Prepares Japanese Marketing Application
JCR is advancing givinostat toward a planned Japanese marketing authorization application by the end of 2026, following the December 2025 exclusive licensing agreement with Italfarmaco. Under the partnership, JCR is responsible for development and commercialization of givinostat in Japan. The company is building on the product’s existing regulatory and clinical experience in other markets while pursuing the requirements for Japanese approval. Givinostat was discovered through Italfarmaco’s research and development activities in collaboration with Telethon and Duchenne Parent Project Italy. The therapy is an orally administered HDAC inhibitor intended to address excessive HDAC activity in DMD muscle. The Japanese orphan drug designation does not constitute marketing approval, and the timing and outcome of any future Japanese regulatory decision will depend on the MHLW review process.
Source :JCR Pharmaceuticals press release
