Osiris becomes trailblazer in stem cell therapy market with Prochymal approval

COLUMBIA, Md.—In what is reportedly the world'sfirst regulatory approval of a manufactured stem cell product, as well as thefirst therapy approved for acute graft-versus-host disease (GvHD) in children,Osiris Therapeutics Inc. announces that it has received market authorizationfrom Health Canada for its stem celltherapy Prochymal (remestemcel-L).

GvHD is a devastating complication of bone marrowtransplantation that kills as many as 80 percent of children affected in severecases, many within just weeks of diagnosis. Osiris says that it is the leadingcause of transplant-related mortality, in which immune cells contained withinthe transplanted marrow recognize the recipient as foreign and mount animmunologic attack. Severe GvHD can cause blistering of the skin, intestinalhemorrhage and liver failure. Even when not fatal, severe GvHD is extremelypainful. Currently, steroids are used as first-line therapy with a success rateof only 30 percent to 50 percent.

When steroids fail, Osiris notes, treatmentoptions 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 foreveryone involved in the responsible development of stem cell therapies," saidDr. C. Randal Mills, president and CEO of Osiris. "Most importantly, today is agreat day for children and their families who bravely face this horrificdisease. While today marks the first approval of a stem cell drug, now that thedoor has been opened, it will surely not be the last."

Prochymal was authorized under Health Canada'sNotice of Compliance with conditions (NOC/c) pathway, which provides access totherapeutic products that address unmet medical conditions and which havedemonstrated a favorable risk/benefit profile in clinical trials. Under theNOC/c pathway, the sponsor must agree to carry out confirmatory clinicaltesting. In Canada, Prochymal is now authorized for the management of acuteGvHD in children who fail to respond to steroids. The approval was based on theresults from clinical studies evaluating Prochymal in patients with severerefractory acute GvHD.

Prochymal is currently available in several countries,including the United States, under an Expanded Access Program. Prochymal isexpected to be commercially available in Canada later this year.

"I am very proud of the leadership role Canada hastaken in advancing stem cell therapy and particularly gratified that thishistoric decision benefits children who would otherwise have little hope," saidDr. Andrew Daly, a clinical associate professor in the Department of Medicineand Oncology at the University of Calgary in Canada and principal investigatorin the Phase III clinical program for Prochymal. "As a result of HealthCanada's comprehensive review, physicians now have an off-the-shelf stem celltherapy in their arsenal to fight GvHD. Much like the introduction ofantibiotics in the late 1920s, with stem cells we have now officially taken thefirst step into this new paradigm of medicine."

Osiris shares rose 14 percent to $6 per share inextended trading after the news was announced.

By the end of this year, Osiris plans to have anapplication into the U.S. Food and Drug Administration for marketing authorization,as well, and Mills anticipates the FDA would apply conditional terms similar tothose of Health Canada.