Osiris Therapeutics, Inc. received regulatory approval to expand patient enrollment into Canada for its ongoing phase III pivotal trial evaluating Prochymal for the treatment of Graft vs. Host Disease.

Graft vs. Host Disease or GVHD is a life threatening immunological reaction that occurs in about 50 per cent of patients who receive donated bone marrow or a similar transplant. GVHD is a form of rejection that occurs when the donated bone marrow attacks the recipient's organs. There is no approved treatment for GVHD. As a result, it is one of the leading causes of death in bone marrow transplant patients.

"We are excited to join this major international effort", said Dr David Allan, assistant prof. of Medicine at the Ottawa Hospital. "In Canada and around the world, there is a definite need for effective therapies to treat GVHD. The initial studies evaluating Prochymal are promising and we look forward to participating in this confirmatory study."

To receive approval to conduct the trial in Canada, Osiris submitted a comprehensive application that included information about the safety and efficacy of the drug, manufacturing and quality specifications, and patient information. The application was reviewed and approved by Health Canada, the federal regulatory agency responsible for the approval of new drugs in Canada.

"This trial is a major undertaking with worldwide implications," said C. Randal Mills, Ph.D., president and CEO of Osiris Therapeutics. "If successful, Prochymal may not only be the first treatment approved for GVHD, but also the first stem cell drug approved for any indication. To accomplish this, we are coordinating an international effort among the world's leading physicians, hospitals, and regulatory agencies. Receiving approval to expand into Canada demonstrates the progress we are making and is a significant milestone for the team."

Prochymal is a preparation of mesenchymal stem cells specially formulated for intravenous infusion. The stem cells are obtained from the bone marrow of healthy adult donors. Prochymal is currently being evaluated for the treatment of GVHD and Crohn's Disease. The phase III trial for GVHD is anticipated to be the final trial before the drug is submitted to FDA, Canadian and European regulatory agencies for full approval. Prochymal has been granted Fast Track status by FDA. The Fast Track programme was established by FDA to accelerate the development of drugs that show promise for treating life threatening conditions. The drug has also been granted Orphan Drug status by FDA. Orphan Drug designation provides incentives to companies that develop drugs for small, underserved patient populations.