Australia's Mesoblast Limited has announced that its licensee in Japan, JCR Pharmaceuticals Co Ltd, has launched its mesenchymal stem cell product TEMCELL HS Injection, for the treatment of acute graft versus host disease (aGVHD) in children and adults in Japan.
TEMCELL is the first allogeneic cell therapy to be fully approved in Japan. The Japanese Government's National Health Insurance set reimbursement for TEMCELL at approximately US$7,700 (¥868,680) per bag of 72 million cells. In Japan, the average adult patient is expected to receive at least 16 or up to 24 bags of 72 million cells. On this basis, Mesoblast expects a treatment course of TEMCELL in an adult Japanese patient to be reimbursed at a minimum of approximately US$123,000 (¥13,898,880) or up to approximately US$185,000 (¥20,848,320).
Under its agreement with JCR, Mesoblast is entitled to receive royalties and other payments at predefined thresholds of cumulative net sales. In the world's largest healthcare market, the United States, there are currently no approved therapies for patients with acute steroid-refractory GVHD, and off-label options have demonstrated mixed efficacy with high toxicity.
To support filing of a biologic license application (BLA) to the United States Food and Drug Administration for regulatory approval, Mesoblast is conducting a 60-patient, open label Phase 3 trial using MSC-100-IV as front-line therapy in children with steroid-refractory aGVHD. After filing a BLA for pediatric approval of MSC-100-IV, Mesoblast plans to conduct a further trial to support a product approval of its cell therapy in adults with gastrointestinal or liver aGVHD, the patient groups who have the highest mortality risk.
In the United States, pricing reimbursement methodology is expected to consider the burden of illness associated with steroid-refractory aGVHD as well as health utilization costs, and may result in a higher price than in Japan.
