NiCord, a novel graft modality developed by Gamida Cell for bone marrow transplants in patients with blood cancers like leukemia and lymphoma, has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA).
The designation is granted to drugs that are intended to treat a serious or life-threatening condition, and where preliminary clinical evidence suggests substantial improvement over existing therapies. It is meant to expedite the development and review of new therapies by giving the company extensive FDA guidance for more efficient and accelerated drug development programs.
“We are very pleased the FDA has recognized the potential of NiCord to address the unmet clinical need in bone marrow transplantation,” Dr. Yael Margolin, president and CEO of Gamida Cell, said in a press release.
“The breakthrough therapy designation creates the foundation for a joint and concerted effort between the FDA and Gamida Cell to bring this important therapy faster to patients. We look forward to continuing our close cooperation with the FDA and other regulatory agencies to a positive conclusion as we prepare for commercialization,” Margolin said.
Each year, more than 60,000 patients with high-risk blood cancers should receive bone marrow transplants, which are currently the only potential cure for these patients.
But to qualify for the treatment, patients require a donor with fully matched tissue typing, which is often difficult to find. As a result, only about half of patients who are supposed to get bone marrow transplants actually receive one.
With NiCord, Gamida Cell is trying to make a difference for patients who can’t find matching donors. NiCord is an alternative to bone marrow transplants that instead uses cells collected from the blood of the umbilical cord. These cells are then cultured using a proprietary NAM platform technology, which enriches the cultured cells in highly functional stem and progenitor cells, as well as dendritic cells.
Gamida Cell believes that NiCord can provide a transplant option that is available to all patients in need, based on results from the pilot and Phase 1/2 studies of NiCord. Compared to unmanipulated umbilical cord blood, the therapy has improved time to neutrophil engraftment (incorporating the transplanted cells into the recipient tissue) from 21 days to 10 days; the number of patients achieving neutrophil engraftment (75% versus 18%); faster platelet engraftment (32 days vs. 46 days); and lower transplant-related mortality (19% vs. 39%).
Additionally, NiCord induced fewer infections and reduced the length of time spent in the hospital.
Gamida Cell is now waiting for positive feedback from the FDA and the European Medicines Agency (EMA) regarding the outline of the Phase 3 trial, after which it will soon begin assessing NiCord in patients with blood cancers.