Osiris
Therapeutics, Inc. (NASDAQ: OSIR), announced today it has received
consent from New Zealand to market its first-in-class stem cell therapy
Prochymal® (remestemcel-L), for the treatment of acute
graft-vs-host disease (GvHD) in children. With this decision New Zealand
joins Canada,
which last month became the world's first internationally recognized
regulatory authority to grant approval to a stem cell drug. Prochymal is
also 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.
"With each of our approvals it becomes clearer that the time for
life-saving stem cell therapies in the practice of medicine has arrived,
and we are humbled to have a leading role," said C. Randal Mills, Ph.D.,
President and Chief Executive Officer of Osiris. "I would like to thank
the professionals at Medsafe for their thoughtful and expeditious review
of this complex application. I would also like to thank the team at
Osiris that continues to do an outstanding job of making Prochymal
available to children around the world suffering from the devastating
effects of GvHD."
Osiris submitted a New Medicine Application (NMA) to Medsafe (New
Zealand's medical regulatory agency) in May of 2011, and was granted
Priority Review in June of 2011. Priority review provides expedited
review for new drugs which offer a significant clinical advantage over
current treatment options. Prochymal was granted provisional consent
under Section 23 of the Medicines Act 1981.
"The incidence of GvHD is likely to rise as the demographic profile of
our transplant population evolves," said Hans Klingemann, M.D., Ph.D., a
Professor of Medicine and the Director of the Bone Marrow &
Hematopoietic Stem Cell Transplant Program at Tufts University School of
Medicine. "Effective strategies to manage the often lethal consequences
of GvHD reduce the overall risk to transplantation and provide the
transplant physician with better options when approaching their most
difficult cases."
Clinical trials have shown that Prochymal is able to induce an
objective, clinically meaningful response in 61-64 percent of children
with GvHD that is otherwise refractory to treatment. Furthermore,
treatment response with Prochymal resulted in a statistically
significant improvement in survival.
"As a mother who watched my son Christian suffer and die from the
horrifying effects of GvHD, while waiting for the regulatory approvals
necessary to allow him access to Prochymal, words cannot express how
happy I am that significant progress is finally being made," said Sandy
Barker, President and Co-founder of the Gold Rush Cure Foundation. "We
are proud to stand side-by-side with Osiris in this historic battle for
our children around the world. Our motto is 'not one more child, not
one more family' and when it comes to GvHD mortality, zero is the
only acceptable number."
Prochymal is now approved in Canada and New Zealand, and is currently
available in seven other countries including the United States under an
Expanded Access Program (EAP). It is expected that Prochymal will be
commercially available in New Zealand later this year.
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