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Bristol Myers cell therapy wins first-of-its-kind approval

Dive Brief:

  • For the first time, the Food and Drug Administration has approved a cell therapy for hard-to-treat forms of two blood cancers.
  • Sold as Breyanzi, the therapy is now cleared for use in certain adults with relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma who have received at least two prior lines of therapy. There is no established standard of care for such patients, so the FDA’s decision to grant an accelerated approval provides a much-needed and potentially transformative option, according to Breyanzi’s developer, Bristol Myers Squibb.
  • The FDA based its decision on results from a clinical trial that evaluated just shy of 120 participants. The trial found that at a certain dose level, around one in five people given Breyanzi had their cancer completely disappear. In total, 45% of participants responded to the therapy, and those responses lasted a median of just under three years.

Dive Insight:

Chronic lymphocytic leukemia and small lymphocytic lymphoma are typically slower progressing and more survivable than other cancers. In fact, treatment often isn’t necessary in the early stages of the disease. Estimates hold that around 85% of people will live at least five years following a diagnosis.

Yet, as these cancers progress, they may require some form of drug intervention. Usually that involves a combination of chemotherapy and targeted medicines like “BTK inhibitors,” a class of drugs that includes AstraZeneca’s Calquence and AbbVie and Johnson & Johnson’s Imbruvica.

For patients who don’t respond or stop responding to those treatments, there are “few options and poor outcomes,” according to Bristol Myers. But the company believes so-called CAR-T cell therapies like Breyanzi can help solve this problem.

Breyanzi is made with a patient’s own white blood cells, which are extracted and then shipped to a manufacturing site. There, they are engineered to better target and destroy malignant “B cells” that express a protein known as CD-19. The souped up blood cells are then sent back to a treatment center where they get reinfused into the patient.

So far, the FDA has approved half a dozen CAR-T therapies, mostly for other blood cancers like follicular lymphoma, multiple myeloma and diffuse large B cell lymphoma. Breyanzi is now the only one indicated for chronic lymphocytic leukemia and small lymphocytic lymphoma.

“For years, attempts to bring other CAR-T cell therapies to patients with relapsed or refractory CLL or SLL met challenges and found little success,” said Bryan Campbell, the commercial head of Bristol Myers’ cell therapy business, in a statement. But following Friday’s approval, “we are now able to offer these patients a personalized option, while further expanding access across the broadest array of B-cell malignancies, to address this critical unmet need.”

Breyanzi is cleared specifically for people who previously received a BTK blocker and a “B-cell lymphoma 2” inhibitor such as AbbVie and Roche’s Venclexta.

The FDA, in granting accelerated approval, noted that Breyanzi’s benefits in these cancers may need to be verified through one or more additional confirmatory studies.

The agency first approved Breyanzi in 2021 as a treatment for adults with relapsed or refractory DLBCL. The drug carries a safety warning for T cell malignancies, neurological side effects, and a kind of immune system reaction called cytokine release syndrome.