JDRF Industry Partner Osiris Receives FDA Orphan Drug Designation for its Diabetes Treatment

JDRF Industry Partner Osiris Receives FDA Orphan Drug Designation for its Diabetes Treatment

A stem cell treatment for people newly diagnosed with type 1 diabetes has been granted “Orphan Drug” status from the U.S. Food and Drug Administration (FDA), speeding the path to market for this promising therapy.

Developed by Osiris Therapeutics, the drug Prochymal is intended to preserve insulin-producing cells in newly diagnosed patients.  At least 30,000 children and adults are diagnosed with type 1 diabetes every year, and could potentially benefit from such a treatment; an estimated 3 million Americans are living with type 1 diabetes. 

The orphan drug designation by the FDA eliminates FDA filing and registration fees and provides tax incentives for as much as 50 percent of the clinical development costs of Prochymal.  The Orphan Drug Act was instituted to promote the development of treatments for underserved patient populations. 

Osiris’ Prochymal is a formulation of “mesenchymal” stem cells – cells derived from adult bone marrow.  It is a novel approach to reversing or halting the autoimmune attack that causes type 1 diabetes.  As part of its Industry Discovery and Development Program, JDRF partnered with the Maryland-based business to support the current Phase II clinical trial, which is evaluating safety and efficacy of Prochymal in people who have beemn recently diagnosed with diabetes.   

Prochymal is also being evaluated as a treatment for other diseases including graft versus host disease (GvHD), Crohn’s Disease, acute myocardial infarction, and pulmonary disease.  Osiris already has received Orphan Drug designation for the drug to treat GvHD.


The announcement by Osiris has several key implications:

  • First, it can speed the development of the drug to help prevent any further destruction of beta cells caused by type 1 diabetes.

  • It is a promising future into further development of treatments that would provide another way to preserve insulin-producing cell function for the newly diagnosed
  • Because of the drug’s extensive safety testing and evaluation for its use with other diseases,  the timetable for testing and approval for its use by people with diabetes could potentially be much shorter, and its development significantly less expensive. 



About JDRF

JDRF is the leading global organization focused on type 1 diabetes (T1D) research. Driven by passionate, grassroots volunteers connected to children, adolescents, and adults with this disease, JDRF is now the largest charitable supporter of T1D research. The goal of JDRF research is to improve the lives of all people affected by T1D by accelerating progress on the most promising opportunities for curing, better treating, and preventing T1D. JDRF collaborates with a wide spectrum of partners who share this goal.

Since its founding in 1970, JDRF has awarded more than $1.7 billion to diabetes research. Past JDRF efforts have helped to significantly advance the care of people with this disease, and have expanded the critical scientific understanding of T1D. JDRF will not rest until T1D is fully conquered. More than 80 percent of JDRF’s expenditures directly support research and research-related education.