FDA Approves First CAR-T Therapy for Rare Lymphoma
For patients with relapsed or refractory marginal zone lymphoma (MZL), treatment options have been limited.
This rare, slow-growing blood cancer often leaves patients with few paths forward after initial therapies fail. But on December 4, 2025, the U.S. Food and Drug Administration (FDA) announced a landmark decision that offers new hope: the approval of Breyanzi (lisocabtagene maraleucel), the first-ever CAR-T cell therapy for this specific type of cancer. [1]
This approval marks a critical milestone, providing a powerful new option for adult patients who have already undergone at least two prior lines of systemic therapy. Breyanzi, developed by Bristol Myers Squibb, is a personalized, one-time treatment that harnesses a patient’s own immune system to fight cancer. [2]
A “Living Drug” with Remarkable Results
CAR-T therapy is a revolutionary approach to cancer treatment. A patient’s T-cells are collected, genetically engineered in a lab to recognize and attack cancer cells, and then infused back into the patient. Breyanzi is a CD19-directed therapy, meaning it’s designed to hunt down and destroy lymphoma cells that have the CD19 protein on their surface. [1]
The clinical trial data that led to this approval is nothing short of extraordinary. In the TRANSCEND FL trial, Breyanzi demonstrated a 95.5% overall response rate (ORR) among 66 evaluable patients. This means that nearly every patient in the trial saw their cancer shrink or disappear. [2]
Clinical Trial Results
Data
Overall Response Rate (ORR)
95.5%
Complete Response (CR)
62.1%
Partial Response (PR)
33.3%
Even more impressive is the durability of these responses. For patients who responded to the treatment, 90.1% were still in remission at the 24-month mark. The median time for a patient to show a response was less than one month. [2]
“The FDA approval of Breyanzi for relapsed or refractory marginal zone lymphoma further solidifies it as the leading CD19-directed CAR T-cell therapy covering the broadest range of B-cell malignancies,” said Lynelle B. Hoch, president of the Cell Therapy Organization at Bristol Myers Squibb. [2]
How Breyanzi Works
The process of creating this “living drug” is a feat of personalized medicine:
Collection: A patient’s T-cells are collected from their blood through a process called leukapheresis.
Engineering: The T-cells are sent to a specialized manufacturing facility where they are genetically modified to produce Chimeric Antigen Receptors (CARs) that target the CD19 protein on cancer cells.
Expansion: The newly engineered CAR-T cells are multiplied into the millions.
Infusion: The CAR-T cells are shipped back to the hospital and infused back into the patient in a single treatment.
Once infused, these cells act as a living medicine, continuously seeking out and destroying lymphoma cells throughout the body.
A New Standard of Care for a Rare Disease
Marginal zone lymphoma has three main subtypes: extranodal, nodal, and splenic. The clinical trial showed that Breyanzi was effective across all three, with overall response rates of 94.1%, 96.9%, and 94.1% respectively. [2]
This approval is not just a win for MZL patients; it also expands Breyanzi’s reach, making it the CAR-T therapy with the broadest range of approved indications. It is also approved for large B-cell lymphoma, follicular lymphoma, and several other B-cell malignancies. [1]
With its remarkable efficacy and durable responses, Breyanzi is set to become a new standard of care for patients with relapsed or refractory marginal zone lymphoma, offering a powerful new weapon in the fight against this rare cancer.
