FDA clears expanded access for Osiris' Prochymal

Osiris Therapeutics has announced the US Food and Drug Administration's clearance to broaden its expanded access program for Prochymal, now  making the investigational stem-cell product available to adults with  life-threatening graft versus host disease (GvHD). In May of 2008, the  FDA approved the first Prochymal EAP for the treatment of pediatric 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 and  is ongoing in another Phase III trial for acute GvHD and in a Phase III  trial for Crohn's disease.