The FDA is requesting Gamida Cell for more data on the company’s wholly owned manufacturing facility before it can go ahead with asking for approval for omidubicel, its cell therapy for blood cancer patients.
The regulatory agency wants to compare omidubicel data from that plant with records from separate facilities that manufactured the drug for the company’s phase 3 study. Gamida, which is backed by Novartis, earlier this year said its registrational trial showed benefits over umbilical cord blood and passed the bar for that study. Omidubicel is formerly known as NiCord.
FDA's request came during a pre-BLA meeting, which takes place before a company files its biologics license application (BLA) to officially ask for approval. During that meeting, the agency didn't ask for “additional clinical data to initiate the BLA (Pre-Biologics License Application) submission once analytical comparability is demonstrated,” Gamida said in a statement.
Gamida now expects to submit the BLA in the first half of 2022 instead of late this year.
Currently, patients with hematological cancers such as leukemia and lymphoma turn to hematopoietic, or bone marrow, stem cell transplantation to restore bone marrow function. However, it can be difficult to source donor-matched cells for all patients, forcing them to use alternatives such as umbilical cord blood. Because an umbilical cord contains fewer stem and progenitor cells, engraftment can take longer, resulting in longer hospital stays and increased risk of infection.
Gamida's aim with omidubicel—an allogeneic, off-the-shelf, hematopoietic stem cell therapy—is to reduce the time to neutrophil engraftment, which results in patients being able to make healthy new cells.