Gamida Cell Provides Regulatory Update on Biologics License Application for Omidubicel
December 14 2020 - 7:00AM
Business Wire
-- 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
Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy
company committed to cures for blood cancers and serious
hematologic diseases, today announced that the company conducted a
Type B Meeting for omidubicel with the U.S. Food and Drug
Administration (FDA) on Friday, December 11, 2020.
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 Gamida Cell generate additional
manufacturing-related data prior to requesting a pre-Biologics
License Application (BLA) meeting. Specifically, the FDA requested
that Gamida Cell demonstrate analytical and clinical comparability
from the company’s planned commercial manufacturing sites. The
company believes the clinical comparability requirement will be met
if the time to neutrophil engraftment in patients from the
company’s ongoing expanded access program (EAP) using omidubicel
produced at Gamida Cell’s planned commercial manufacturing sites is
consistent with the results achieved in the Phase 3 clinical trial.
Based on the feedback received at the meeting with FDA, Gamida Cell
intends to submit a full BLA for omidubicel in the second half of
2021 in lieu of the company’s previous plan to initiate a rolling
BLA submission by the end of 2020. The revised plan could support a
potential commercial launch of omidubicel in the United States as
early as mid-2022.
“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 Julian Adams, Ph.D., chief
executive officer of Gamida Cell. “With this clarity, we look
forward to proceeding towards a full BLA submission in the second
half of 2021. We continue to prepare for the commercialization of
omidubicel on the revised timeline, as we continue to provide
omidubicel to patients in need through our expanded access
program.”
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 U.S. and EU.
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.
About Omidubicel
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 U.S. Food and Drug
Administration or any other health authority.
About GDA-201
Gamida Cell applied the capabilities of its NAM-based cell
expansion technology to develop GDA-201, an innate natural killer
(NK) cell immunotherapy for the treatment of hematologic and solid
tumors in combination with standard of care antibody therapies.
GDA-201 addresses key limitations of NK cells by increasing the
cytotoxicity and in vivo retention and proliferation in the bone
marrow and lymphoid organs of NK cells expanded in culture. GDA-201
is in Phase 1 development through an investigator-sponsored study
in patients with refractory non-Hodgkin lymphoma and multiple
myeloma.1 For more information on the clinical study of GDA-201,
please visit www.clinicaltrials.gov.
GDA-201 is an investigational therapy, and its safety and
efficacy has not been established by the U.S. Food and Drug
Administration or any other health authority.
About Gamida Cell
Gamida Cell is an advanced cell therapy company committed to
cures for patients with blood cancers and serious blood diseases.
We harness our cell expansion platform to create therapies with the
potential to redefine standards of care in areas of serious medical
need. For additional information, please visit www.gamida-cell.com
or follow Gamida Cell on LinkedIn or Twitter at @GamidaCellTx.
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 Securities and Exchange Commission (SEC) on February 26,
2020, its Reports on Form 6-K filed with the SEC on May 18, 2020,
August 11, 2020 and November 10, 2020, and other filings that
Gamida Cell makes with the SEC from time to time (which are
available at http://www.sec.gov), the events and circumstances
discussed in such forward-looking statements may not occur, and
Gamida Cell’s actual results could differ materially and adversely
from those anticipated or implied thereby. Any forward-looking
statements speak only as of the date of this press release and are
based on information available to Gamida Cell as of the date of
this release.
1 Clinicaltrials.gov identifier NCT03019666
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