Delivering good news to patients with malignant blood cancer, innovative stem cell transplant products are one step closer to approval

▎WuXi AppTec Content Team Editor On February 9, 2022, Gamida Cell announced that it has initiated rolling submission of the Biologics License Application (BLA) for the stem cell/bone marrow transplant product omidubicel to the U.
S.
FDA
.

Omidubicel is a potentially life-saving treatment for patients with blood cancers who require stem cell transplants
.

The company expects to complete the BLA submission in the second quarter of 2022
.

According to the press release, omidubicel is expected to be the first advanced cell therapy product approved by the U.
S.
FDA for allogeneic stem cell transplantation
.

For patients with malignant blood cancers who are eligible for an allogeneic stem cell transplant, the procedure is the best chance for a potential cure
.

Unfortunately, many patients are still unable to find a suitable donor each year
.

Compared to other donor sources, omidubicel has a looser matching criteria and has the potential to improve outcomes for patients with allogeneic stem cell transplants and allow access to treatment for patients who cannot find a suitable donor
.

Omidubicel is an allogeneic hematopoietic stem cell (bone marrow) transplant solution for the treatment of blood cancer patients
.

Utilize the epigenetic regulation function of the small molecule nicotinamide (NAM) to improve the expansion of cells in vitro while preserving their identity and function
.

The press release mentions that omidubicel is the first stem cell transplant product to receive breakthrough therapy designation from the U.
S.
FDA, and has also received orphan drug designation in the U.
S.
and the European Union
.

A Phase 3 clinical trial evaluating its efficacy and safety met its primary endpoint, demonstrating significantly shorter time to neutrophil colonization in patients in the omidubicel arm compared to control patients who received standard cord blood transplantation A key milestone in recovery from transplantation
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The trial also met its secondary endpoints of decreased time to platelet colonization, fewer infections and shorter hospital stays in patients
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▲The development of Omidubicel (Image source: Gamida Cell's official website) Dr.
Julian Adams, CEO of Gamida Cell, said: "We are delighted to have reached this important milestone for omidubicel and to bring this potential therapy to the needy sooner.
patients
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In Phase 3 clinical trials, omidubicel demonstrated statistically significant reductions in neutrophil colonization time and hospital stay, infection risk, and platelet colonization time
.

Based on these positive data, we believe omidubicel is expected to address the same Existing unmet need in allogeneic transplantation, providing a new standard of care with an opportunity to treat more patients
.

” Reference: [1] Gamida Cell Initiates Rolling Submission of Biologics License Application for Omidubicel.
Retrieved February 9, 2022 , from https://investors.
gamida-cell.
com/news-events/press-releases/news-release-details/gamida-cell-initiates-rolling-submission-biologics Disclaimer: WuXi AppTec content team focuses on introducing global Advances in Biomedical Health Research
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This article is for information exchange purposes only, and the views expressed in this article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's support or opposition to the views expressed in this article
.

This article is also not a treatment plan recommendation
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For guidance on treatment options, please visit a regular hospital
.