World’s first stem cell drug approved in Canada
pharmafile | May 21, 2012 | News story | Sales and Marketing | Canada, regenerative medicine, stem cell
Canada has approved the world’s first stem cell drug, marking an historic day for the burgeoning therapy.
Osiris’ Prochymal is now approved in Canada in children with graft versus host disease (GvHD), a potentially deadly complication from a bone marrow transplant, when newly implanted cells attack the patient’s body.
Prochymal is made up of bone marrow stem cells derived from an adult donor and is designed to control inflammation, promote tissue regeneration and prevent scar formation – GvHD affects around 3,500 to 4,000 worldwide.
“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.”
The approval is based on recent trial results that showed statistically significant improvement in survival in patients taking Prochymal, when compared with a historical control population of children with refractory GvHD.
Andrew Daly, principal investigator in the Phase III programme for Prochymal, said: “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 1920s, with stem cells we have now officially taken the first step into this new paradigm of medicine.”
It has not been an easy road for the firm – in 2009, two late-stage clinical trials failed to show the drug was more effective overall than a placebo in treating the disease.
But it did show promise in certain subgroups of patients, and the firm later re-analysed its data to confirm that its drug could increase survival in this patient population.
The Canadian regulators have told Osiris that it must carry out further testing on the drug after it reaches the market.
Osiris, which is based in Columbia, Maryland, plans to submit its drug with the FDA by the end of this year, including its newly analysed information.
Boost for stem cell research
Dozens of adult stem cell therapies are currently moving through clinical trials, and Canada’s approval of Prochymal will be a boost to many working in the field.
But the use of stem cells remains controversial, as some are derived from the cells of embryos.
However, stem cells derived from adult tissue such as fat or bone marrow – such as with Osiris’ drug – can get around these ethical concerns.
Osiris signed a pact potentially worth around $1.4 billion with Genzyme in 2008 to develop Prochymal and another stem cell drug, Chondrogen, for osteoarthritis.
But in February Genzyme, now a part of Sanofi, said it had discontinued its project with Prochymal for GvHD.
Ben Adams
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