FDA Grants Breakthrough Designation Therapy for Novel Bone Marrow Transplant Graft Modality

The U.S. Food and Drug Administration (FDA) granted breakthrough designation therapy for NiCord®, a novel graft modality for hematopoietic cell transplants in patients with hematologic malignancies. NiCord is an ex vivo expanded cell graft that uses nicotinamide platform technology, which expands cells from the umbilical cord blood. NiCord is in development as an alternative to a hematopoietic cell transplant for patients who cannot find a donor with fully matched tissue.

The FDA’s decision was based on data from an international, multicenter, phase I/II study. Sixteen patients who were 12 to 65 years old with high-risk hematologic malignancies were included in the study. They received a transplant with NiCord as a standalone graft following myeloablative therapy. One year after transplant, patient outcomes were compared with outcomes from a control group of 125 similar patients enrolled in the Center for International Blood and Marrow Transplant Research registry who were transplanted with cord blood.

Patients who received a transplant with NiCord experienced a 101-fold increase in the number of CD34+ cells compared with patients who received a transplant with unmanipulated cord blood. Those in the NiCord group also demonstrated faster neutrophil engraftment (10 days vs. 21 days p<0.0001) and faster platelet engraftment (median = 32 vs. 46 days; p<0.001) compared with controls.

At 16 days post-transplant, 75 percent of NiCord recipients achieved neutrophil engraftment compared with 18 percent in the control group (p<0.0001), and at 42 days post-transplant, 56 percent versus 27 percent, respectively, achieved platelet engraftment (p=0.015).

One-year transplant-related mortality was 19 percent for NiCord and 39 percent for the control group (p=0.12).

An international, multicenter, phase III study of NiCord is set to begin before the end of the year.

Sources: Gamida Cell press release, October 11, 2016; Gamida Cell press release, June 6, 2016.

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