Courier | A boon for patients with malignant blood cancer, innovative stem cell transplant products are one step closer to approval
On February 9, 2022, Gamida Cell announced that it has initiated a rolling application for the licensing of biological products (BLA) for stem cell/bone marrow transplant product omidubicel to the U.S. FDA. Omidubicel is used as a potentially life-saving therapeutic for the treatment of blood cancer patients who require stem cell transplants. The company is on track to complete the BLA submission in the second quarter of 2022.
The press release states that omidubicel is expected to become the first advanced cell therapy product approved by the US FDA for allogeneic stem cell transplantation. For patients with malignant blood cancer who are eligible for allogeneic stem cell transplantation, this procedure is the best chance of obtaining a potential cure. Unfortunately, many patients still can't find a suitable donor each year. Omidubicel's matching criteria are more relaxed compared to other donor sources, with the potential to improve outcomes for allogeneic stem cell transplant patients and to enable treatment of suitable donor patients who cannot be found.
Omidubicel is an allogeneic hematopoietic stem cell (bone marrow) transplantation solution for the treatment of blood cancer patients. The epigenetic regulation of small molecule nicotinamide (NAM) is used to improve the amplification effect of in vitro cultures while preserving cell characteristics and function. The press release mentions that omidubicel is the first stem cell transplant product to be recognized by the US FDA as a breakthrough therapy, and has also been qualified as an orphan drug in the US and the European Union. A Phase 3 clinical trial evaluating its efficacy and safety achieved the primary endpoint, demonstrating a significant reduction in neutrophil colonization time compared to control patients who received standard cord blood transplantation, a key milestone in patient recovery from stem cell transplantation. The trial also met a secondary endpoint, which reduced the patient's platelet colonization time, reduced infections, and shortened hospital stays.
▲ Omidubicel's research and development progress (Source: Gamida Cell official website)
Dr Julian Adams, CEO of Gamida Cell, said: "We are delighted to reach this important milestone of omidubicel and to make this potential therapy hopefully faster for patients in need. In a Phase 3 clinical trial, omidubicel demonstrated a statistically significant reduction in neutrophil colonization time and length of hospital stay, a reduction in the risk of infection, and a reduction in platelet colonization time. Based on these positive data, we believe that omidubicel holds promise to address the existing unmet needs in allogeneic transplantation, provide a new standard of treatment, and have the opportunity to treat more patients. ”
Resources:
[1] Gamida Cell Initiates Rolling Submission of Biologics License Application for Omidubicel. Retrieved February 9, 2022, from 网页链接
