FDA clears Gamida’s cord blood therapy for cancer patients

The FDA has approved a stem cell-based therapy for cancer patients from Israeli biotech Gamida Cell that is designed to help them recover more quickly from aggressive chemotherapy.

Omisirge (omidubicel) – based on stem cells harvested from donor umbilical cord blood – has been cleared for use in patients aged 12 and over who are undergoing haematological stem cell transplantation (HSCT) for serious blood cancers.

Prior to an HSCT, patients have to undergo high-intensity, myeloablative chemo and/or radiotherapy to destroy their bone marrow and prepare the way for the transplanted cells, which leaves them vulnerable to infections and other complications.

Omisirge, which is Gamida Cell’s first approved product, is given as a single intravenous dose and is intended as a treatment for patients who are unable to find a matched stem cell transplant donor.

The company’s chief executive – Abbey Jenkins – said that the product could expand the number of patients eligible for an HSCT and help improve patient outcomes.

In clinical trials, Omisirge was shown to return levels of the infection-fighting neutrophil white blood cells to normal ranges within 12 days of dosing, which compares to 22 days for a standard cord blood transplant.

That also translated into reduced infection rates, with 39% of patients in the Omisirge arm and 60% of patients in the standard cord blood arm developing a grade 2 or 3 fungal infection in the first 1,200 days after their transplant.

Gamida Cell also noted that 40% of patients recruited into its phase 3 trial were racially and ethnically diverse, which could help address disparities in access to stem cell transplants.

“The approval of Omisirge is a significant development in haematopoietic stem cell transplantation,” said Steven Devine, chief medical officer at the National Marrow Donor Program in the US.

“Patients who are Black or African American have just a 29% chance of finding a match via the donor registry versus a 79% chance for patients who are White,” he pointed out.

For now, Gamida Cell is keeping mum on its launch and pricing plans for Omisirge, other than to say it will be delivered to transplant centres within 30 days of the start of manufacturing at its facility in Kiryat Gal, Israel.

In 2021, the FDA asked for additional data on how the commercial material made at the plant compared with the material used in clinical trials, which delayed Gamida Cell’s filing for the product.

The company – which was forced into a restructuring programme last month as it held on for FDA approval – has indicated that it will seek a commercial partner to sell and distribute the therapy in the US. It has set a target of treating around 2,500 patients with Omisirge annually by 2027.