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FDA grants orphan drug status for Targazyme’s TZ101 for use with regulatory T cells

The US Food and Drug Administration (FDA) has granted Orphan Drug designation to Targazyme's fucosyltransferase enzymes and small molecule product, TZ101, to prevent and reduce the severity and incidence of graft versus host disease (GVHD) in patients eligible for hematologic stem cell transplant.

The company said that TZ101 could potentially transform hematopoietic stem cell transplantation by reducing patient morbidity and mortality from GVHD.

GVHD is a serious, life-threating complication of stem cell transplantation and is very difficult to manage clinically.

Targazyme chairman and chief executive officer Lynnet Koh said: "The granting of Orphan Drug status for TZ101 for prevention of GVHD in stem cell transplant patients, as well as our previous Orphan Drug designation of TZ101 for cord blood transplantation, provides additional validation of our innovative platform technologies.

"TZ101 and our second product, TZ102 are enabling technologies for improving efficacy outcomes for multiple cell-based therapeutic approaches used to prevent and treat a variety of different diseases for which there is a high unmet medical need.

"In addition to initiating our registration trial with TZ101 in hematopoietic stem cell transplantation, we plan to embark on our cancer immunotherapy trial later this year."

Both TZ101 and TZ102 are used at the point-of-care to treat therapeutic cells immediately before infusion into the patient using a simple procedure that is easily incorporated into existing medical practice.