Gamida Cell wins cell therapy first in severe aplastic anaemia from FDA

Gamida Cell has won a new approval from the US Food and Drug Administration (FDA) for Omisirge (omidubicel-onlv), making it the first hematopoietic stem cell transplant (HSCT) therapy licensed for severe aplastic anaemia.

The Ayrmid company’s novel transplant option, which is derived from umbilical cord blood, is now authorised for patients aged six years and older following reduced intensity conditioning.

Dr Joe Wiley, Ayrmid Chairman and Chief Executive Officer, said: “Alongside its initial FDA approval in haematological malignancy in 2023, Omisirge is a new and novel transplant option for the many severe aplastic anaemia patients who do not have a matched related donor.

“This approval is potentially transformative for patients living with [severe aplastic anaemia], given the rapid and sustained recovery of blood counts observed.”

The cell therapy addresses the limitations of umbilical cord blood as a source, including delayed haematopoietic recovery and increased infections and provides additional graft options for these patients who require HSCT. Clinical data showed no reported cases of BMT-CTN severe acute graft-versus-host disease (GVHD).

Dr Megha Kaushal, Acting Deputy Director of the CBER Office of Therapeutic Products and paediatric haematologist, said: “Omisirge will shorten time to neutrophil recovery which leads to shorter recovery times after transplant and may improve infection rates in this patient population.”

Clinical efficiency of Omisirge

FDA’s approval was based on data from an ongoing study evaluating Omisirge in patients six years old, which illustrated early and sustained neutrophil engraftment in 12 of 14 patients in the efficacy population. Median time to neutrophil recovery was 11 days.  

Furthermore, data showed a “rapid” immune recovery post-transplant as well as a disease-free and overall survival rate of 92 percent.

Dr Vinay Prasad, Chief Medical and Scientific Officer and Director of the FDA’s Center for Biologics Evaluation and Research, said: “This approval is revolutionary in the therapeutic landscape and fundamentally changes how we approach treatment for severe aplastic anaemia, where earlier treatment has potential to alter one’s life course.”