Osiris Therapeutics Inc.
) recently received good news with its stem cell therapy,
Prochymal, gaining approval in Canada. Health Canada granted
approval to Prochymal for the treatment of acute graft-vs-host
disease (GvHD) in children who do not respond to steroids.
This makes Prochymal the first manufactured stem cell product to
gain approval and the first treatment to gain approval for
The regulatory process in Canada was not exactly smooth with
Prochymal failing to gain first-round approval. Last year, Health
Canada had informed Osiris that it had a few questions on the
candidate. The agency had also raised questions regarding
post-marketing commitments. Osiris filed a complete response to the
inquiries from the Canadian regulatory authority following which an
independent expert advisory panel voted in favor of approving
Prochymal has been approved under Health Canada's Notice of
Compliance with conditions (NOC/c) pathway. Through this pathway,
patients are able to access treatments which target unmet medical
conditions and which have a favorable risk/benefit profile in
clinical studies after evaluating its safety and efficacy.
When a product gains approval under the NOC/c pathway, it means
that the company will be required to conduct confirmatory clinical
studies. Osiris will be conducting a case matched confirmatory
study as part of the conditions for gaining approval. Patients
being treated with Prochymal will be encouraged to participate in
the study which will keep track of the long-term effects of
Osiris intends to launch Prochymal commercially in Canada later
this year. We note that Prochymal is available under an Expanded
Access Program (EAP) in eight countries including the U.S.
Prochymal's approval in Canada is a milestone for Osiris.
Prochymal is the lead candidate at Osiris and represents
significant commercial potential. Prochymal is currently being
evaluated for other indications as well including refractory
Crohn's disease (phase III), type I diabetes, and myocardial
infarction (heart attack). Results from the phase II myocardial
infarction study should be out in the second quarter of 2012.
We currently have low visibility on the status of Osiris'
development and commercialization agreement with Genzyme, a
) company, for Prochymal. Earlier this year, Sanofi, in its fourth
quarter press release, had said that it has discontinued the
development of Prochymal for GvHD. Osiris said that the
announcement was made without its knowledge or advice and clarified
that Prochymal's development has not been discontinued. Although
Osiris had not received any communication from Sanofi regarding the
termination of their agreement, Osiris notified Sanofi that it is
treating Sanofi's statement as an intention to terminate the
According to Osiris, all rights to Prochymal will return to
Osiris without the company being required to compensate Sanofi.
Osiris believes it can now pursue commercialization agreements for
Prochymal with other parties.
However, following this announcement, Sanofi informed Osiris
that it is not in agreement with Osiris' interpretation of its
fourth quarter R&D update. Osiris has asked Sanofi for a
clarification regarding the matter. We await more information on
the status of the Sanofi deal.
OSIRIS THERAPTC (OSIR): Free Stock Analysis
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