In May 2008, the FDA approved the first Prochymal expanded access program for the treatment of pediatric graft versus host disease (GVHD) patients.
Congress and the FDA created the expanded access program to facilitate the availability of promising new drugs to desperately ill patients before general marketing begins. The program allows for investigational drugs to be made available to patients under certain circumstances during evaluation in late stage clinical trials when no satisfactory alternative therapy is available.
For expanded access, the FDA must determine that the available scientific evidence, taken as a whole, demonstrates that the drug may be effective and would not expose the patients to unreasonable risks. Additionally, the FDA permits companies meeting certain criteria to charge for the investigational product. Expanded access to Prochymal was initially restricted to only pediatric patients suffering from steroid refractory GVHD until patient enrollment in the Phase III pivotal trial was completed.
Prochymal, a formulation of adult mesenchymal stem cells designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration and preventing scar formation, is in Phase III clinical trials for GVHD and Crohn’s disease. Enrollment in the Phase III pivotal trial for steroid-refractory GVHD was recently completed. Enrollment is ongoing in another Phase III trial for acute GVHD and in a Phase III trial for Crohn’s disease.
In November 2008, Osiris and Genzyme announced a strategic alliance for the development and commercialization of Prochymal. Under the terms of the agreement, Osiris will commercialize Prochymal in the US and Canada, and Genzyme will commercialize the treatment in all other countries.