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Health Canada approves marketing of company's stem cell drug for GVHD.

Osiris Therapeutics Inc. (OSIR), a Columbia, Md.-based stem cell company, on May 17 announced that Health Canada has approved the marketing of the stem cell therapy Prochymal (remestemcel-L) for the treatment of acute graft-vs.-host disease (GvHD) in children.

The decision marks the world's first regulatory approval of a manufactured stem cell product and the first therapy approved for GvHD, a devastating complication of bone marrow transplantation that kills up to 80 percent of children affected, many within just weeks of diagnosis.

Shares of the drug maker jumped 15 percent to $6.05 after hours. Through the close, the stock is up 7.1 percent over the past three months.

Prochymal has had its share of clinical failures. In two late-phase clinical trials conducted in 2009, Prochymal showed almost no therapeutic benefit over a placebo in treating graft-vs.-host disease.

French pharmaceutical firm Sanofi announced in February that it would no longer pursue development of the therapy for markets outside North America.

But Osiris has found that the drug is effective in treating severe forms of GvHD in children.

GvHD is die 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. Steroids are used as first-line therapy with a success rate of only 30-50 percent.

When steroids fail, treatment options are limited to immunosuppressive agents used off-label with little benefit and significant toxicities.

"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," said Andrew Daly, M.D., clinical associate professor, department of medicine and oncology at die University of Calgary, Canada, and principal investigator in the phase 3 clinical program for Prochymal. "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."

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.

Health Canada's audiorization was made following die recommendation of an independent expert advisory panel, commissioned to evaluate Prochymal's safety and efficacy.

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 demonstrated a clinically meaningful response at 28 days post initiation of therapy in 6164 percent of patients treated. Furthermore, treatment with Prochymal resulted in a statistically significant improvement in survival when compared to a historical control population of pediatric patients with refractory GvHD p=0,028).

The survival benefit was most pronounced in patients with the most severe forms of GvHD. As a condition of approval, the clinical benefit of Prochymal will be further evaluated in a case matched confirmatory trial and all patients receiving Prochymal will be encouraged to participate in a registry that will monitor the long-term effects of the therapy.

"Refractory GvHD is not just deadly to the patients it afflicts, but is devastating for the family, friends, and caregivers who watch helplessly as the disease progresses," said Joanne Kurtzberg, M.D., head of die pediatric bone marrow transplant program at Duke University and lead investigator for Prochymal. "I have personally seen Prochymal reverse the debilitating effects of severe GvHD in many of my patients and now, after nearly two decades of research, the data demonstrating consistently high response rates, a strong safety profile and improved survival clearly support the use of Prochymal in the management of refractory GvHD."

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

Prochymal is an intravenous formulation of mesenchymal stem cells (MSCs) derived from the bone marrow of healthy adult donors between die 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 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 currently approved in Canada for the management of acute graft-versus-host disease (GvHD) in children. Prochymal is currently in phase 3 trials for refractory Crohn's disease and also being evaluated in clinical trials for the treatment of myocardial infarction (heart attack) and type 1 diabetes.

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Title Annotation:Sales & Marketing
Publication:Stem Cell Lab World
Geographic Code:1CANA
Date:May 21, 2012
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