Bristol Myers Squibb Gets FDA Priority Review for T-Cell Therapy
The FDA priority review for the T-cell therapy comes four months after the company received a Refusal to File letter regarding the Biologics License Application for the same therapy.
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By - Bristol Myers Squibb and bluebird bio, Inc. recently announced that FDA accepted their request for a priority review for Idecabtagene vicleucel, a chimeric antigen receptor (CAR) T-cell therapy for the treatment of adult patients with multiple myeloma.
Idecabtagene vicleucel (Ide-cel) is specifically intended for patients who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody.
FDA’s Prescription Drug User Fee Act (PDUFA) goal date is March 27, 2021.
“Today’s Priority Review milestone recognizes the potential of this first anti-BCMA CAR T cell therapy to address a critical unmet need of patients with multiple myeloma,” Stanley Frankel, MD, senior vice president of cellular therapy development at Bristol Myers Squibb, said in the announcement.
“We are pleased by the significant progress that is being made in partnership with patients and the multiple myeloma community to bring ide-cel to adults with relapsed and refractory multiple myeloma who are triple-class exposed and may benefit from an important new therapeutic option.”
T-cell therapy is a form of immunotherapy that uses specially altered T cells, part of the immune system, to fight cancer.
Researchers collect a sample of a patient's T cells from their blood and modify it to produce special structures called CAR on their surface.
Ide-celbinds to BCMA on the surface of multiple myeloma cells leading to CAR T cell growth, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells, Bristol Myers Squibb noted.
The Biologic License Application is based on results from the Phase 2 KarMMa study that evaluated the safety and efficiency of Ide-cel in 128 adults with heavily pre-treated and highly refractory multiple myeloma.
Previously, Ide-cel was granted Breakthrough Therapy Designation (BTD) by FDA. The European Medicines Agency also gave it PRIority MEdicines (PRIME) designation and validation.
“Today’s acceptance of the BLA for ide-cel for Priority Review by the FDA marks a key moment in our journey to bring this BCMA-directed CAR T cell therapy to multiple myeloma patients who are in desperate need of new options,” said Joanne Smith-Farrell, PhD, chief operating officer oncology of bluebird bio.
“Based on the body of evidence we have generated in an advanced, heavily pre-treated patient population, our confidence in the potential of ide-cel as an important treatment option remains high. Together with our partners at Bristol Myers Squibb, we are committed to continue working with the FDA to deliver this promising therapy to patients in an expeditious manner.”
This announcement comes four months after FDA issued a Refusal to File letter to Bristol Myers Squibb and bluebird bio regarding the Biologics License Application for ide-cel.
The BLA was submitted in March 2020 but FDA determined that the chemistry, manufacturing and control module of the BLA required further detail to complete the review after a preliminary assessment.
Additionally, Bristol Myers Squibb and bluebird bio’s clinical development program for ide-cel includes clinical studies in earlier lines of treatment for patients with multiple myeloma, including newly diagnosed multiple myeloma.
“Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment,” Bristol Myers Squibb said in the May announcement.
“Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.”
Other pharmaceutical companies continuing to further their immunotherapy research is Gilead, Kit, and oNKo-innate, who formed a partnership to support the discovery and development of next-generation cancer immunotherapies back in late April.
The immunotherapy approaches will focus on T cell mediated anti-tumor immunotherapy, including checkpoint inhibition and CAR T cell therapy.
