FDA Approves First Cell-Based Gene Therapy for Adults With Multiple Myeloma

The U.S. Food and Drug Administration has approved Abecma® (idecabtagene vicleucel) to treat adults with relapsed (returning) or refractory (does not respond to treatment) myeloma. Abecma is for those who have had at least 4 prior different types of treatment and have not responded to treatment or whose disease has returned. This makes Abecma the first gene therapy approved for multiple myeloma.1

Multiple myeloma is an uncommon kind of blood cancer that can affect many bones in the body. It can also cause low blood counts, damage the bones and kidneys, and weaken the immune system. Myeloma accounted for nearly 2 percent of new cancer cases in the United States in 2020. The approval of Abecma gives people who have this type of cancer a new treatment option.1

The FDA granted Abecma 2 special designations:1

  • Orphan Drug – This gives incentives to encourage drug development for rare diseases like multiple myeloma
  • Breakthrough Therapy – This means the FDA helps speed up the development and review of drugs that treat serious or life-threatening conditions

Abecma is currently only available through a restricted program called the Risk Evaluation and Mitigation Strategies (REMS). This is because Abecma carries the risk of serious side effects that can be fatal or life-threatening.1

What are the ingredients in Abecma?

The active ingredient is Abecma is idecabtagene vicleucel.3

How does Abecma work?

Abecma is an immunotherapy drug known as a chimeric antigen receptor (CAR) T-cell therapy. It works by sticking to B-cell maturation antigens (BCMA). These are proteins that are found in cancerous cells found in people with multiple myeloma. When Abecma attaches to BCMAs, it sends a signal to activate your immune system. This helps your immune system find and kill cancer cells.3

Every Abecma dose is customized using the person’s own T-cells. The T-cells are collected and then genetically modified, including a new gene that helps to target and kill cancer cells. The cells, once modified, are infused back into the person.1

Evidence for Abecma

Abecma was approved based on the results of the phase 2 KarMMa clinical trial. The trial included 127 people with relapsed (returning) or refractory (does not respond to treatment) myeloma who had received at least 3 or 4 other prior treatments.1

Results showed:1

  • 72 percent of the study participants had a partial or complete response. This means their cancer shrank or disappeared after treatment with Abecma.
  • 28 percent of the study participants had a complete response. This means their cancer disappeared after treatment with Abecma. Of this group, 65 percent maintained this response for at least 1 year.

What are the possible side effects of Abecma?

The most common side effects of Abecma include:1,3

  • Fatigue
  • Fever, chills
  • Nausea, diarrhea
  • Decreased appetite
  • Headache
  • Dizziness, lightheadedness
  • Confusion
  • Difficulty speaking, slurred speech
  • Cough
  • Difficulty breathing
  • Fast or irregular heartbeat

Abecma has a boxed warning, the strictest warning from the FDA, because it may cause severe side effects. These serious side effects can be fatal or life-threatening and include:1-3

  • Cytokine release syndrome (CRS), which is an immune system reaction that can cause fever, nausea, headache, rash, rapid heartbeat, low blood pressure, and trouble breathing
  • Damage to the brain or nervous system
  • Hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS), which can cause low blood pressure, low oxygen levels, low blood cell levels, and problems with the kidneys and other organs
  • Low levels of blood cells, leading to bleeding and infection

If you have any side effects that may be life-threatening, contact 911 right away.

These are not all the possible side effects of Abecma. Talk to your doctor about what to expect or if you experience any changes that concern you during treatment with Abecma.

Things to know about Abecma

People taking Abecma should be monitored for signs of severe side effects at least once a day for 7 days following treatment with the drug.3

People with active infections or inflammatory disorders should not take Abecma.3

There is not enough information about Abecma to know if this drug is safe to use when pregnant or breastfeeding. Before using this drug, tell your doctor if you are pregnant, plan to become pregnant, or breastfeeding.3

Before beginning treatment for multiple myeloma, tell your doctor about all your health conditions and any other drugs, vitamins, or supplements you are taking. This includes over-the-counter drugs.

For more information, read the full prescribing information for Abecma.

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