Gamida Cell Provides Regulatory Update on Biologics License Application for Omidubicel
-- Feedback from FDA supports proceeding to BLA Submission for omidubicel with additional manufacturing requirements; the company plans to submit full BLA in second half of 2021
During the meeting, the FDA provided encouraging feedback regarding the Phase 3 study of omidubicel pertaining to the pre-specified primary and secondary endpoints. The FDA also recommended that
“Although we are disappointed by the delay in timing to bring omidubicel to patients after a potential FDA approval, we are encouraged by the FDA’s reaction to our Phase 3 data as the pivotal trial of omidubicel achieved pre-specified primary and secondary endpoints. Commercial manufacturing readiness activities are underway, and we believe we will be able to complete all the requirements discussed with FDA,” said
The planned BLA submission for omidubicel is supported by a Phase 3 study which met its primary and secondary endpoints. The international, multi-center, randomized Phase 3 study was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing a bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. Omidubicel is the first bone marrow transplant cell therapy product to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the
As previously announced, given the encouraging Phase 1 data presented at the 2020 ASH meeting, the company also plans to submit an IND for GDA-201, its expanded natural killer cell investigational therapy, in 2021.
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). In both Phase 1/2 and Phase 3 clinical studies (NCT01816230, NCT02730299), omidubicel demonstrated rapid and durable time to engraftment and was generally well tolerated. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn®, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the
GDA-201 is an investigational therapy, and its safety and efficacy has not been established by the
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of and data reported from the clinical trials of Gamida Cell’s product candidates, anticipated regulatory filings including BLA submission, launch readiness and FDA approval, commercialization efforts and Gamida Cell’s expectations regarding its projected ongoing operating activities, which statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; and clinical, scientific, regulatory and technical developments. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 20-F, filed with the
1 Clinicaltrials.gov identifier NCT03019666
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