On October 28, 2012 REGiMMUNE Corporation reported that RGI-2001, the company’s liposomal formulation of alpha-GalCer, has been granted Orphan Drug Designation by the U.S. Food & Drug Administration (FDA) for the treatment of Graft-versus-Host Disease (GvHD) associated with hematopoietic stem cell transplantation (Press release, REGimmune, OCT 28, 2012, View Source [SID1234642237]).
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Orphan Drug Designation is a special status granted by FDA to a novel product to treat a rare disease or condition on the basis of Orphan Drug Act (ODA) In order to give incentive to biotech companies to tackle treatments for race diseases, the company of the product is qualified for some benefits such as tax credit or waiver of a prescription drug user fee.
About RGI-2001
RGI-2001 is a liposomal formulation of alpha-GalCer (a CD1d ligand), which induces regulatory T cells (Treg), a key player in immune tolerance. Because the compound promotes transplantation tolerance, Treg has been shown to have significant potential for treating Graft-versus-Host Disease (GvHD). In studies by independent researchers, Treg has proven to produce longer patient survival because it reduces rejection without reducing an anti-tumor graft versus leukemia (GvL) effect. Unlike immunosuppressents that destroy entire T cell subsets, RGI-2001 induces Treg while maintaining normal immune cell functions and has the potential to reduce GvHD and improve survival for patients.
About Graft-versus-Host Disease (GvHD)
GvHD is a serious complication that results from rejection of the donor Human Stem Cell Transplantation (HSCT). GvHD is caused by the donor’s immune system recognizing the cells/organs derived from the patient (recipient) as pathogens (foreign body). A low grade of GvHD is regarded as positive, because it reflects a normal immune reaction while trying to eliminate residual cancer cells. However, a more severe grade of GvHD can be life threatening as it may significantly damage skin cells, fast growing cells in the gastrointestinal (GI) tract and liver cells.