Italfarmaco S.p.A. and JCR Pharmaceuticals Co., Ltd., listed on the Tokyo Stock Exchange under the code 4552, have announced an exclusive agreement granting JCR the rights to develop and commercialize givinostat in Japan. This agreement gives JCR the sole authority to market givinostat for the treatment of Duchenne muscular dystrophy and manage all local clinical development and regulatory approval processes.
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Givinostat, marketed as Duvyzat in the United States, United Kingdom, and European Union, is an oral histone deacetylase inhibitor designed to treat DMD regardless of the specific dystrophin gene mutation. The drug has received regulatory approval in multiple major markets, although it is not yet approved for use in Japan. Beyond commercialization, the partnership also establishes a strategic collaboration to explore additional opportunities in rare disease research and development.
Antonio Nardi, Vice President and Head of Business and Portfolio Development at Italfarmaco, emphasized the significance of the collaboration, stating that this partnership represents an important step in expanding access to givinostat while enhancing the company’s presence in the rare disease sector. He highlighted JCR’s local expertise, innovative approach, and patient-focused strategy as key reasons why the company is an ideal partner for global rare disease initiatives.
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Shin Ashida, Chairman, President, and CEO of JCR Pharmaceuticals, expressed that working with Italfarmaco is a strategic milestone for the company and could serve as the foundation for long-term collaboration. He underlined JCR’s ongoing dedication to developing therapies for patients with rare diseases and noted that givinostat will broaden treatment options for the DMD community in Japan.
Francesco De Santis, Chairman of Italfarmaco Group, added that the collaboration with JCR allows the companies to expand access to a critical therapy for Japanese patients with Duchenne muscular dystrophy while laying the groundwork for future innovations in rare disease treatments. He stressed the shared commitment to delivering meaningful solutions where patients need them most.
Duchenne muscular dystrophy is a rare, progressive neuromuscular disorder caused by mutations in the dystrophin gene. These mutations prevent the formation of functional dystrophin, which is essential for maintaining the dystrophin-associated protein complex in muscle fibers. The absence of dystrophin causes increased levels of histone deacetylase in muscle cells, impairing the activation of genes needed for muscle repair. This leads to ongoing muscle damage, chronic inflammation, poor regeneration, and eventual replacement of muscle tissue with scar tissue and fat. DMD primarily affects boys, with symptoms usually appearing between ages two and five. The condition gradually worsens, causing muscle weakness, loss of the ability to walk, and eventually impacting cardiac and respiratory muscles, which are the leading causes of premature death. In Japan, DMD affects approximately 3,500 patients, and globally, it occurs in about one in every 5,050 boys.
This collaboration marks a major step forward in making innovative treatments like givinostat more accessible to patients in Japan and demonstrates the shared commitment of Italfarmaco and JCR to addressing unmet needs in rare diseases.
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