Orphan Designation Provides 7-Year Post Approval Marketing Exclusivity, Tax Credits and Elimination of FDA Prescription Drug User Fees SAN DIEGO, CA--(Marketwired - February 10, 2015) - Targazyme Inc., a clinical-stage biopharmaceutical company developing enzyme technologies and products to improve efficacy outcomes for stem cell transplantation, immunotherapy, gene therapy and regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to TZ101 to prevent and reduce the severity and incidence of graft vs. host disease (GVHD) in patients eligible for hematologic stem cell transplant. GVHD is a serious, life-threating complication of stem cell transplantation.Orphan drug status confirms the importance of Targazyme's novel treatment approach to prevent and reduce the incidence and severity of GVHD in patients with blood cancers where stem cell transplant is prescribed.TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD, which occurs in a large percentage of these patients and is very difficult to manage clinically. "Our work with TZ101 demonstrates impressive increases in the persistence and activity of regulatory T cells in preclinical models of GVHD," said Dr. Elizabeth J. Shpall, Deputy Chair of the Department of Stem Cell Transplantation and Cellular Therapy … Continue reading
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