Osiris Therapeutics Inc. announced it has received market authorization from Health Canada to market its stem cell therapy Prochymal(R) (remestemcel-L), for the treatment of acute graft-vs-host disease (GvHD) in children. The historic 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.
"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 Andrew Daly, M.D., Clinical Associate Professor, Department of Medicine and Oncology at the University of Calgary, Canada and Principal Investigator in the phase 3 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 1920's, 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.
"Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies," said C. Randal Mills, Ph.D., President and Chief Executive Officer 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."
Health Canada's authorization was made following the 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 61-64 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, MD, Head of the 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.
"Today Osiris turns the promise of stem cell research into reality, delivering on decades of medical and scientific research," said Peter Friedli, Chairman and Co-founder of Osiris. "It took 20 years of hard work and perseverance and I want to personally thank everyone involved for their dedication to this important mission."
In addition to the extensive intellectual property protection Osiris has around Prochymal, which includes 48 issued patents, Health Canada's decision will also provide Prochymal with regulatory exclusivity within the territory. Canada affords eight years of exclusivity to Innovative Drugs such as Prochymal, and an additional six-month extension is available since it addresses a pediatric population.