Osiris Therapeutics Inc. (OSIR) 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 GvHD.
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.
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 Prochymal.
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 Sanofi (SNY) 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 agreement.
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.
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