- FDA Rejects Bristol Myers Cell Squibb’s CAR T-Cell Therapy
- Bristol Myers Squibb Gives $53M to Support Cancer Immunotherapies
- Gilead, Kite Ink Deal with oNKo-innate for Cancer Immunotherapies
The RMAT designation program helps to advance the development of therapies for chronic conditions. Breyanzi is the first regenerative medicine therapy with RMAT designation to be licensed by the FDA.
“Today’s approval represents another milestone in the rapidly progressing field of gene therapy by providing an additional treatment option for adults with certain types of cancer affecting the blood, bone marrow, and lymph nodes,” Peter Marks, MD, PhD, director of the FDA’s Center for Biologics Evaluation and Research, said in the announcement.
“Gene and cell therapies have evolved from promising concepts to practical cancer treatment regimens,” Marks continued.
FDA based its approval on the safety and efficacy of Breyanzi, which was established in a multicenter clinical trial of over 250 adults with refractory or relapsed large B-cell lymphoma.
The trial found that the complete remission rate after treatment with Breyanzi was 54 percent.
Each dose of Breyanzi is a customized treatment created using a patient’s own T-cells. The patient’s T cells are collected and then genetically modified to include a new gene that helps to target and kill lymphoma cells, FDA stated.
Once the cells are genetically modified, they are infused back into the patient.
Cytokine release syndrome (CRS) is a systematic response to the activation and proliferation of CAR-T cells. Generally, CRS causes high fever, flu-like symptoms, and neurologic toxicities, which can be life-threatening.
Therefore, FDA approved Breyanzi with a risk evaluation and mitigation strategy (REMS) due to the risk of CRS and neurologic toxicities. Side effects will usually appear within the first few weeks after treatment, but some side effects may happen later.
FDA is requiring healthcare facilities that administer Breyanzi be specially certified and that any staff involved in the prescribing, dispensing, or administering of the drug be trained to recognize and manage the risks of CRS and neurologic toxicities.
Additionally, the manufacture must conduct a post-marketing observational study involving patients treated with Breyanzi.
B-cell lymphoma is the most common type of non-Hodgkin lymphoma in adults, with nearly 77,000 new cases of non-Hodgkin lymphoma diagnosed in the US each year, FDA said. B-cell lymphoma alone represents about one in three newly diagnosed cases.
Bristol Myers Squibb acquired the novel CAR T cell therapy as part of its January 2019 acquisition of Celgene.
However, the approval of the drug has faced multiple delays, calling into question the financial benefits of the $74 billion merger deal.
FDA also issued a Refusal to File letter to Bristol Myers Squibb and Bluebird Bio in May regarding the Biologics License Application (BLA) for idecabtagene vicleucel, a chimeric antigen receptor CAR-T cell immunotherapy for patients with multiple myeloma.
Idecabtagene vicleucel (Ide-cel) was the first CAR-T cell therapy submitted for regulatory approval to target multiple myeloma and Bristol Myers Squibb acquired the drug through its merger deal with Celgene.
The recent FDA approval for the company’s CAR-T cell therapy, Breyanzi, is a step in the right direction and could mean that the deal between Bristol Myers Squibb and Celgene is paying off.
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