FDA approves for the first time a commercial 'expanded' umbilical cord blood product

The US Food and Drug Administration (FDA) approves cell therapy for patients with blood cancers to reduce the risk of infection after stem cell transplantation.

The FDA approved Omisirge, a substantially modified allogeneic umbilical cord cell therapy to speed the recovery time of neutrophils , a subset of white blood cells, in the body and reduce the risk of infection.

"Today's approval is an important advance in cell therapy treatment of patients with blood cancers," said Peter Marks, MD, Ph.D., director of the FDA's Center for Biologics Evaluation and Research. “Speeding the return of the body's white blood cells may reduce the chance of severe or overwhelming infection associated with stem cell transplantation. This approval reflects the FDA's continued commitment to supporting the development of innovative therapies for life-threatening cancers."

Important points of this cell therapy:

Designed for adult and pediatric patients 12 years of age and older with blood cancer who were planning to undergo cord blood transplantation after a myeloablative conditioning regimen ( treatment such as radiation therapy or chemotherapy).
Omisirge, administered as a single intravenous dose, is composed of allogeneic human stem cells from umbilical cord blood that are processed and cultured with nicotinamide (a form of vitamin B3).
Each dose of Omisirge is patient specific. This contains healthy stem cells from a prescreened allogeneic donor, meaning it comes from a different person rather than using the patient's own cells.
The safety and effectiveness of Omisirge were supported by a randomized multicenter study comparing Omisirge transplantation with umbilical cord blood transplantation in subjects 12 to 65 years of age.
Omisirge is the product of "expanded" umbilical cord blood, which means that the umbilical cord blood has been cultured in a laboratory to selectively increase the number of cells to compensate for the limited number of hematopoietic stem cells in a unit of cord blood. umbilical cord.
It brings together the best characteristics for which transplant specialists are inclined to choose bone marrow or umbilical cord blood as a graft source for a stem cell transplant.
Omisirge engrafts much faster than bone marrow, with a median neutrophil engraftment time (ANC>500) of 10 days, compared to 15-17 days for bone marrow.

This approval marks a before and after for the biotechnology sector.

Fountain:

https://parentsguidecordblood.org/en/news/1st-fda-approval-omisirge-expanded-cord-blood

https://www.fda.gov/news-events/press-announcements/fda-approves-cellular-therapy-for-patients-with-blood-cancers-to-reduce-risk- of