Osiris Therapeutics, Inc., a leading stem cell company, has received approval from New Zealand to market its first-in-class stem cell therapy Prochymal (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. With this decision New Zealand joins Canada, which last month became the world’s first internationally recognized regulatory authority to grant approval to a stem cell drug.

Prochymal is also the first therapy approved for GvHD - a devastating complication of bone marrow transplantation that kills up to 80 per cent of children affected, many within just weeks of diagnosis.

“With each of our approvals it becomes clearer that the time for life-saving stem cell therapies in the practice of medicine has arrived, and we are humbled to have a leading role,” said C Randal Mills, PhD, president and chief executive officer of Osiris. “I would like to thank the professionals at Medsafe for their thoughtful and expeditious review of this complex application. I would also like to thank the team at Osiris that continues to do an outstanding job of making Prochymal available to children around the world suffering from the devastating effects of GvHD.”

Osiris submitted a New Medicine Application (NMA) to Medsafe (New Zealand's medical regulatory agency) in May of 2011, and was granted Priority Review in June of 2011. Priority review provides expedited review for new drugs which offer a significant clinical advantage over current treatment options. Prochymal was granted provisional consent under Section 23 of the Medicines Act 1981.

“The incidence of GvHD is likely to rise as the demographic profile of our transplant population evolves,” said Hans Klingemann, MD, PhD, a Professor of Medicine and the Director of the Bone Marrow & Hematopoietic Stem Cell Transplant Programme at Tufts University School of Medicine. “Effective strategies to manage the often lethal consequences of GvHD reduce the overall risk to transplantation and provide the transplant physician with better options when approaching their most difficult cases.”

Clinical trials have shown that Prochymal is able to induce an objective, clinically meaningful response in 61-64 per cent of children with GvHD that is otherwise refractory to treatment. Furthermore, treatment response with Prochymal resulted in a statistically significant improvement in survival.

“As a mother who watched my son Christian suffer and die from the horrifying effects of GvHD, while waiting for the regulatory approvals necessary to allow him access to Prochymal, words cannot express how happy I am that significant progress is finally being made,” said Sandy Barker, President and Co-founder of the Gold Rush Cure Foundation. “We are proud to stand side-by-side with Osiris in this historic battle for our children around the world. Our motto is 'not one more child, not one more family' and when it comes to GvHD mortality, zero is the only acceptable number.”

Prochymal is now approved in Canada and New Zealand, and is currently available in seven other countries including the United States under an Expanded Access Program (EAP). It is expected that Prochymal will be commercially available in New Zealand later this year.

GvHD represents a major unmet medical need with no approved treatment until Prochymal. GvHD is the leading cause of transplant related mortality, in which immune cells contained within the transplanted marrow recognize the recipient as foreign and mount an immunologic attack. Severe GvHD can cause blistering of the skin, intestinal hemorrhage and liver failure. Severe GvHD is extremely painful and fatal in up to 80 percent of cases. Currently, steroids are used as first-line therapy with a success rate of only 30-50 per cent. When steroids fail, treatment options are limited to immunosuppressive agents used off-label with little benefit and significant toxicities.

Prochymal is the world’s first approved drug with a stem cell as its active ingredient. Developed by Osiris Therapeutics, Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs), which are derived from the bone marrow of healthy adult donors between the ages of 18 and 30 years. The MSCs are selected from the bone marrow and grown in culture so that up to 10,000 doses of Prochymal can be produced from a single donor. Prochymal is truly an off-the-shelf stem cell product that is stored frozen at the point-of-care and infused through a simple intravenous line without the need to type or immunosuppress the recipient. Prochymal is approved in Canada and New Zealand for the management of acute graft-versus-host disease (GvHD) in children and is available for adults and children in eight countries including the United States, under an Expanded Access Programme. Prochymal is currently in Phase 3 trials for refractory Crohn’s disease and is also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes.

Osiris Therapeutics, Inc. is the leading stem cell company, having developed the world’s first approved stem cell drug, Prochymal. The company is focused on developing and marketing products to treat medical conditions in inflammatory, cardiovascular, orthopedic and wound healing markets.