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Osiris becomes trailblazer in stem cell therapy market with Prochymal approval
COLUMBIA, Md.—In what is reportedly the world's first regulatory approval of a manufactured stem cell product, as well as the first therapy approved for acute graft-versus-host disease (GvHD) in children, Osiris Therapeutics Inc. announces that it has received market authorization from Health Canada for its stem cell therapy Prochymal (remestemcel-L).
GvHD is a devastating complication of bone marrow transplantation that kills as many as 80 percent of children affected in severe cases, many within just weeks of diagnosis. Osiris says that it 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. Even when not fatal, severe GvHD is extremely painful. Currently, steroids are used as first-line therapy with a success rate of only 30 percent to 50 percent.
When steroids fail, Osiris notes, treatment options have thus far been limited to immunosuppressive agents used off-label "with little benefit and significant toxicities."
"Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies," said Dr. C. Randal Mills, president and CEO of Osiris. "Most importantly, today is a great day for children and their families who bravely face this horrific disease. While today marks the first approval of a stem cell drug, now that the door has been opened, it will surely not be the last."
Prochymal was authorized under Health Canada's Notice of Compliance with conditions (NOC/c) pathway, which provides access to therapeutic products that address unmet medical conditions and which have demonstrated a favorable risk/benefit profile in clinical trials. Under the NOC/c pathway, the sponsor must agree to carry out confirmatory clinical testing. In Canada, Prochymal is now authorized for the management of acute GvHD in children who fail to respond to steroids. The approval was based on the results from clinical studies evaluating Prochymal in patients with severe refractory acute GvHD.
Prochymal is currently available in several countries, including the United States, under an Expanded Access Program. Prochymal is expected to be commercially available in Canada later this year.
"I am very proud of the leadership role Canada has taken in advancing stem cell therapy and particularly gratified that this historic decision benefits children who would otherwise have little hope," said Dr. Andrew Daly, a clinical associate professor in the Department of Medicine and Oncology at the University of Calgary in Canada and principal investigator in the Phase III clinical program for Prochymal. "As a result of Health Canada's comprehensive review, physicians now have an off-the-shelf stem cell therapy in their arsenal to fight GvHD. Much like the introduction of antibiotics in the late 1920s, with stem cells we have now officially taken the first step into this new paradigm of medicine."
Osiris shares rose 14 percent to $6 per share in extended trading after the news was announced.
By the end of this year, Osiris plans to have an application into the U.S. Food and Drug Administration for marketing authorization, as well, and Mills anticipates the FDA would apply conditional terms similar to those of Health Canada.