Multiple Myeloma Drug Approved for Relapsed or Refractory Types

(HealthDay News) — The U.S. Food and Drug Administration has approved Abecma (idecabtagene vicleucel) as a personalized CAR T-cell therapy for triple-class exposed relapsed or refractory multiple myeloma.

The approval is for the treatment of adult patients with relapsed or refractory multiple myeloma after two or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is a one-time infusion, with a new recommended dose range of 300 to 510 x 106 CAR-positive T cells. Boxed warnings for Abecma include cytokine release syndrome, neurologic toxicities, hemophagocytic lymphohistiocytosis/macrophage activation syndrome, prolonged cytopenia, and secondary hematological malignancies.

The approval was based on data from the phase 3 KarMMa-3 trial of 254 patients. At a median follow-up of 15.9 months, Abecma more than tripled the primary end point of progression-free survival versus standard regimens, with a median progression-free survival of 13.3 months versus 4.4 months (hazard ratio, 0.49). Overall response rates were also significantly improved with Abecma, with 71 percent of patients treated with Abecma achieving a response and 39 percent achieving a complete or stringent complete response compared with 42 and 5 percent, respectively, of those receiving standard regimens. Responses to Abecma were durable, with a median duration of response of 14.8 months (or 20 months for patients who derived a complete response or better).”With this approval, these patients now have an opportunity to be treated at an earlier line of therapy with a potentially transformative therapy that offers significantly improved progression-free survival for this difficult-to-treat disease that has had no established treatment approach,” Al-Ola A. Abdallah, M.D., chair of the U.S. Myeloma Innovations Research Collaborative, said in a statement.

Approval of Abecma was granted to Bristol Myers Squibb and 2seventy bio.

What is multiple myeloma?

Multiple myeloma is a cancer of the plasma cells, a type of white blood cell found in bone marrow. These plasma cells normally produce antibodies to fight infection. In myeloma, however, abnormal plasma cells multiply uncontrollably, crowding out healthy cells and producing abnormal proteins that can damage organs.

Multiple myeloma can cause a variety of symptoms, but some of the most common include:

Bone pain: This is a frequent symptom, as myeloma cells can weaken bones and cause fractures. Back and rib pain are common, but any bone can be affected.

Anemia: Myeloma crowds out healthy blood cells, leading to a shortage of red blood cells. This can cause fatigue, weakness, and shortness of breath.

Frequent infections: A lack of healthy white blood cells makes it harder to fight infection.

Kidney problems: Abnormal proteins produced by myeloma cells can damage the kidneys.

High blood calcium: Myeloma can cause the release of too much calcium into the blood, leading to confusion, nausea, and constipation.

Bleeding problems: A decrease in platelets, another type of blood cell, can cause easy bruising, nosebleeds, and bleeding gums.
It’s important to note that these symptoms can also be caused by other conditions. If you are experiencing any of these, it’s important to see a doctor to get a diagnosis.

How multiple myeloma affects Black Americans

This complex disease disproportionately affects Black Americans.

Americans are about twice as likely to be diagnosed with multiple myeloma compared to white Americans. They also tend to be diagnosed at a younger age, with a median of four to five years earlier. This earlier onset can be critical, as younger patients may have other health demands and may not be eligible for all treatment options.

The exact reasons for the higher incidence in Black Americans are still under investigation. Some possibilities include:

Genetic factors: Certain genetic variations may play a role, but more research is needed

Socioeconomic factors: Disparities in access to healthcare, healthy food options, and quality housing may contribute.
Precursor conditions: African Americans are more likely to have a precursor condition called monoclonal gammopathy of undetermined significance (MGUS), which can develop into myeloma.

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