Scimitar Equity Blog

The Biologics and Genetic Therapies Directorate of Health Canada has completed its initial evaluation and accepted for full review OSIR’s New Drug Submission (NDS) of Prochymal (remestemcel-L),

 an adult stem cell therapy for the treatment of graft vs. host disease (GvHD). Based on a separate review of summary clinical data, Health Canada has notified Osiris that the application has been granted Priority Review, shortening the examination period from 300 to 180 days.

• If successful, Prochymal would become the world’s first approved stem cell therapy.

Priority Review is granted when Health Canada determines that the summary clinical data provided demonstrates substantial evidence of the drug’s effectiveness in treating a life-threatening condition. Priority Review candidates are subject to the same quality, safety and efficacy requirements as non-priority submissions, but are processed more quickly.

• The application is the 1st in OSIR’s global registration strategy for Prochymal. The NDS was filed in the electronic Common Technical Document (eCTD) format to facilitate review and allow for efficient submission in other territories. The submission is also the first application for full approval of a stem cell therapy anywhere in the world,
• As part of the review, Health Canada has also informed Osiris of its plans to add Prochymal to its Register of Innovative Drugs subject to a final review upon approval. Registration confers eight years of market exclusivity beginning on the date of Prochymal’s approval, during which no submission for a generic version of Prochymal will be approved.

Prochymal (remestemcel-L) is a preparation of mesenchymal stem cells (MSCs) formulated for intravenous infusion.

• The MSCs utilized in Prochymal are isolated from the bone marrow of healthy young adult donors, avoiding the controversy surrounding embryonic and fetal cell sources. They are grown in culture, permitting large-scale production. Because the cells can be expanded, thousands of doses can be produced from a single donation. Studies indicate that MSCs are able to down-regulate severe inflammation and work at the cellular level to rebuild damaged tissue through the coordinated release of tissue-specific growth factors.

Prochymal is being evaluated in clinical programs for the treatment of Crohn’s disease, acute myocardial infarction, type 1diabetes and pulmonary disease.

• Prochymal has been granted Fast Track status by the FDA for GvHD and Crohn’s disease, and is the first stem cell product to receive FDA expanded access approval, making the product available now to patients with life-threatening GvHD. Prochymal also obtained Orphan Drug status for GvHD and type 1 diabetes from the FDA as well as Orphan Drug status for GvHD from the European Medicines Agency.