Stem Cell Treatment for Type 1 Diabetes Receives FDA Orphan Drug Designation


Osiris Therapeutics, Inc. announced that it has been granted Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for Prochymal as a treatment for type 1 diabetes mellitus. The FDA instituted the Orphan Drug Act to promote the development of treatments for underserved patient populations. To be eligible for Orphan Drug designation, the treatment must target a disease that affects fewer than 200,000 new patients per year in the United States.

Orphan Drug designation provides market exclusivity for up to seven years following approval, eliminates FDA filing and registration fees, and provides tax incentives for as much as 50 percent of the clinical development costs.

Prochymal, a formulation of adult mesenchymal stem cells (MSC) designed to provide therapeutic benefit by controlling inflammation, promoting tissue regeneration, and preventing scar formation, is currently being evaluated in a double-blind, placebo-controlled Phase II clinical trial for type 1 diabetes. Osiris is developing Prochymal in partnership with the Juvenile Diabetes Research Foundation (JDRF) as a treatment for patients with newly diagnosed type 1 diabetes mellitus.

In type 1 diabetes, the patient’s own immune system attacks and destroys insulin-producing islet cells in the pancreas, resulting in the loss of blood-sugar control. Currently, there are no approved treatments for altering the rate of destruction of these critical islet cells, called beta cells. Preclinical studies first conducted by researchers at Genzyme found that MSCs may delay the progression of type 1 diabetes by preserving beta cell function.

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Beth Tully
Beth Tully
10 years ago

My son developed adult onset of Type I diabetes. How do we find out about adult stem cell studies that he might qualify to be involved with?

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