Photo courtesy of NHS
The US Food and Drug Administration (FDA) has granted breakthrough therapy designation for NiCord®, a product in development as a graft modality for patients undergoing hematopoietic stem cell transplant to treat high-risk hematologic malignancies.
NiCord consists of cells from a single unit of umbilical cord blood cultured in nicotinamide (a vitamin B derivative) and cytokines that are typically used for expansion (thrombopoietin, interleukin 6, FLT3 ligand, and stem cell factor).
Data from the pilot and phase 1/2 studies of NiCord have suggested the therapy can provide a clinically meaningful improvement in time to neutrophil engraftment over traditional cord blood transplant.
Additional research presented at EBMT 2016 showed that patients who received NiCord had fewer infections, shorter hospital stays, quicker platelet engraftment, and improved non-relapse mortality when compared to patients who received a traditional cord blood transplant.
The phase 3 registration study of NiCord is scheduled to begin before the end of this year.
In addition to breakthrough designation, NiCord also has orphan designation from the FDA as a treatment for patients with acute lymphoblastic leukemia, acute myeloid leukemia, Hodgkin lymphoma, or myelodysplastic syndromes.
NiCord is being developed by Gamida Cell.
About breakthrough designation
The FDA’s breakthrough designation is intended to expedite the development and review of new therapies for serious or life-threatening conditions.
Breakthrough designation entitles the company developing a therapy to more intensive FDA guidance on an efficient and accelerated development program, as well as eligibility for other actions to expedite FDA review, such as a rolling submission and priority review.
To earn breakthrough designation, a treatment must show encouraging early clinical results demonstrating substantial improvement over available therapies with regard to a clinically significant endpoint, or it must fulfill an unmet need.
