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news.Osiris Therapeutics has received clearance from the FDA to initiate an expanded access treatment program for Prochymal, making the investigational stem cell product available to children with life-threatening graft versus host disease.
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Under the expanded access program, children two months to 17 years in age inclusive with grades B-D graft versus host disease (GvHD) not responsive to steroids are eligible for treatment.
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, FDA must determine that the available scientific evidence, taken as a whole, demonstrates that the drug may be effective for its intended use or would not expose the patients to unreasonable and significant additional risk of illness or injury. Additionally, FDA permits companies meeting certain criteria to charge for the investigational product.
Prochymal, a formulation of adult mesenchymal stem cells administered through a standard intravenous line, is currently in Phase III clinical trials, the final stage of clinical testing before submission to FDA for marketing approval.