February 24, 2016 8:17am
First Allogeneic Cell therapy product launched in Japan by Mesoblast Licensee for the treatment of acute graft versus host disease (aGVHD) in children and adults – BUY;
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 ¥868,680 (approximately US$7,700) per bag of 72 million cells. In Japan, the average adult patient is expected to receive at least 16 or up to24 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 ¥13,898,880 (approximately US$123,000) or up to ¥20,848,320 (approximately US$185,000).
· 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 US, there are currently no approved therapies for patients with acute steroid-refractory GVHD, and off-label options have demonstrated mixed efficacy with high toxicity.
The Bottom Line: To support filing of a biologic license application (BLA) to the US FDA for regulatory approval, MESO is conducting a 60-patient, open label P3 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.
MESO closed at $6.07 – BUY, news is fodder for appreciation.
Mesoblast is developing MSC-100-IV for the treatment of aGVHD following an allogeneic bone marrow transplant (BMT). In patients who have received a BMT, donor cells may attack the recipient (the person receiving the transplant), causing aGVHD, resulting in activation of pro-inflammatory T-cells and tissue damage in the skin, gut and liver which is often fatal.
According to the Center for International Blood and Marrow Transplant Research, there are approximately 30,000 allogeneic BMTs globally per year for diseases including hematological cancers, with 25% of all cases in the pediatric population. Nearly 50% of all allogeneic BMT patients develop aGVHD.
Liver or gastrointestinal involvement occur in up to 40% of all patients with aGVHD and are associated with the greatest risk of death, with mortality rates of up to 85%.
