The U.S. Food and Drug Administration (FDA) has granted approval to StemCyte's biologics license application (BLA) for REGENECYTE, a hematopoietic progenitor cell (HPC) therapy derived from human umbilical cord blood. This approval marks StemCyte as the first commercial biotech company to receive such a license for this specific cell therapy type, offering a new option for patients with certain blood and immune system disorders.
Indication and Use
REGENECYTE is an allogeneic hematopoietic stem cell therapy approved for use in unrelated donor hematopoietic progenitor cell transplantation. It is indicated for patients with inherited or acquired hematopoietic system disorders, including those resulting from myeloablative treatments. The therapy is administered alongside an appropriate preparative regimen to facilitate immunologic and hematopoietic reconstitution.
Clinical Trial Data
The FDA's approval was based on data from multiple studies, including the COBLT clinical trial (Study 1), a retrospective review of literature and observational studies (Study 2), and data from the Center for International Blood and Marrow Transplant Research (Study 3). Key findings include:
- Study 1: Among 324 patients, 76% achieved neutrophil recovery by Day 42 (95% CI: 71%, 81%), 57% achieved platelet recovery by Day 100 (95% CI: 51%, 63%), and 65% achieved erythrocyte recovery by Day 100 (95% CI: 58%, 71%). The median time to neutrophil recovery was 27 days, to platelet recovery was 90 days, and to erythrocyte recovery was 64 days.
- Study 2: Among 1299 patients, 77% achieved neutrophil recovery by Day 42 (95% CI: 75%, 79%) and 45% achieved platelet recovery by Day 100 (95% CI: 42%, 48%). The median time to neutrophil recovery was 25 days and to platelet recovery was 122 days.
- Study 3: Among 54 patients, 91% achieved neutrophil recovery by Day 42 (95% CI: 81%, 97%) and 72% achieved platelet recovery by Day 100 (95% CI: 58%, 83%). The median time to neutrophil recovery was 22 days and to platelet recovery was 50 days.
Safety Profile
The most common infusion-related adverse reactions associated with REGENECYTE include hypertension, vomiting, nausea, bradycardia, and fever. Warnings and precautions include hypersensitivity reactions, infusion reactions, graft versus host disease, engraftment syndrome, graft failure, malignancies of donor origin, transmission of serious infections, and transmission of rare genetic diseases. The product is contraindicated for patients with known sensitivity to dimethyl sulfoxide (DMSO), Dextran 40, or plasma proteins.
StemCyte's Future Plans
StemCyte is exploring the potential of its HPC, Cord Blood products for other therapeutic applications, including aging-related diseases, chronic fatigue syndrome, and acute stroke. The company is currently conducting Phase II clinical trials for the treatment of long COVID syndrome and acute stroke.
Dr. Tong Young Lee, CEO of StemCyte, stated, "With one of the largest inventories of cord blood stem cells worldwide, StemCyte is uniquely positioned to supply, manufacture, and develop advanced cell therapy products. Looking ahead, we are committed to driving the development of innovative therapies, achieving sustainable growth through diversified business strategies, and solidifying our position as a global leader in the field of cell therapy."
