There is great news for parents suffering the harrowing ordeal of their child needing a bone marrow transplant. The announcement came from Osiris Therapeutics Inc. (NASDAQ: OSIR) on Friday that Health Canada has approved its groundbreaking stem cell therapy Prochymal® (remestemcel-L). The drug can be used to treat children with acute graft-vs-host disease. (GvHD).

The decision is a historic one, as it’s both the first stem cell drug going into formal use, as well as the first treatment for GvHD. The disease is a devastating breakdown occurring after a bone marrow transplant and kills around 80% of children affected, often within a matter of weeks.

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 confirmed :

“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 … 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.”

The approval process for Prochymal was implemented under Health Canada’s Notice of Compliance with conditions (NOC/c) pathway. The basis of the procedure allows a new drug to come onto the market where there are unmet medical needs. The approval is granted with the provision that the drug has demonstrated risk / reward benefits in previous clinical trials and that the manufacturer agrees to undertake additional confirmatory clinical testing.

C. Randal Mills, Ph.D., President and Chief Executive Officer of Osiris confirmed his’ companies happiness at being able to help conquer the disease :

“Today is not only a great day for Osiris, but for everyone involved in the responsible development of stem cell therapies … 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.”

Where children with GvHD are not responding to treatment with steroids, which is presumably most of them, the use of Prochymal will now be authorized. Health Canada based it’s approval on previous clinical studies of the drug, in which 64% of patients showed results; the survival rate compared to historical data was drastically improved, even in patients with severe cases. Additional clinical evaluation of Prochymal now will be undertaken, including enrolling patients in a registry to discover any long term effects.

Joanne Kurtzberg, MD, Head of the Pediatric Bone Marrow Transplant Program at Duke University and Lead Investigator for Prochymal

“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 … 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. Peter Friedli, Chairman and Co-founder of Osiris concludes:

“Today Osiris turns the promise of stem cell research into reality, delivering on decades of medical and scientific research … It took 20 years of hard work and perseverance and I want to personally thank everyone involved for their dedication to this important mission.”

Osiris has 48 patents protecting Prochymal, and Health Canada’s have agreed to provide Prochymal with regulatory exclusivity within their territory. Canada affords eight years of exclusivity to Innovative Drugs, such as Prochymal, with an additional six-month extension because it addresses a pediatric disease. Parents, doctors and shareholders can all rest easy.

Written by Rupert Shepherd.