Is Abecma an orphan drug?

Abecma is considered an orphan drug because it is used to treat multiple myeloma, a rare disease. Orphan drugs are used to treat, prevent or diagnose rare “orphan” diseases that affect fewer than 200,000 people in the U.S. About 36,000 people are expected to be diagnosed with multiple myeloma in 2024.

Why is Abecma an Orphan Drug?

Abecma was granted Orphan Drug status which provides an incentive to manufacturers to develop drugs for rare “orphan” diseases, affecting fewer than 200,000 people in the U.S. On March 27, 2021 the FDA approved Celgene's Abecma (idecabtagene vicleucel) for the treatment of adults with multiple myeloma.

Drugs for rare diseases may not be developed due to a small patient populations and the possibility of low profits for the pharmaceutical manufacturer. Orphan drug designation by the FDA increases the chances that a drug company will research and market orphan drugs for rare diseases.

Learn more: What is an orphan drug?

What is Abecma approved to treat?

Abecma (idecabtagene vicleucel) is a a B-cell maturation antigen (BCMA)-directed T cell immunotherapy approved for the treatment of adults with multiple myeloma who have received 2 or more different treatment regimens that either did not work or stopped working. These treatments include an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody.

Multiple Myeloma

Multiple myeloma is a rare blood cancer that develops in the bone marrow, the spongy part of the inside of your bone.

• Your bone marrow is where white blood cells, red blood cells, and platelets (that help with blood clotting) are made.
• In multiple myeloma, there is uncontrolled growth of plasma cells, one type of white blood cell that makes antibodies to help you fight off infections.
• The myeloma cells leave no room for growth of other healthy blood cells. The cancer cells can also invade the outer layers of the bone and weaken them, which may lead to bone fractures.

Multiple myeloma tends to occur in older people, developing around the age of 60. Common symptoms due to multiple myeloma include:

• bone pain
• headaches
• increased risk for infections
• extreme tiredness / fatigue
• kidney damage
• peripheral neuropathy (nerve pain that may cause a “pins and needles” feeling)
• easy bruising
• loss of appetite
• nausea / vomiting

How does Abecma work in multiple myeloma?

Abecma works by recognizing and binding to B-cell maturation antigen (BCMA), a protein that is found on normal cells and almost all cancer cells in multiple myeloma. Binding leads to the death of BCMA-expressing myeloma cells.

Abecma is known as a chimeric antigen receptor (CAR) T cell immunotherapy.

• It is made from a patient’s own white blood cells that are then genetically modified to help fight multiple myeloma.
• It's is given as a one-time infusion (into a vein in your arm) in a REMS-certified healthcare facility.
• You will need to spend some time in the facility to receive the therapy, and then stay close to the facility for several weeks or more to be monitored by your healthcare providers.

Abecma was the first B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell immunotherapy approved by the FDA.

How well does Abecma work?

In a Phase 2 clinical study of 100 patients, 72% of patients partially or completely responded, with 28% showing a complete response (a disappearance of all signs of multiple myeloma tumors). A complete response does not mean the myeloma has been cured. Not all patients will respond to treatment with Abecma.

In addition, in the Phase 3 KarMMa-3 pivotal study, 254 patients were randomized to receive Abecma and 132 patients received standard combination regimens for multiple myeloma based on investigator discretion.

Participants had relapsed and refractory multiple myeloma who had received two to four prior lines of treatment, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody, and were refractory to the last treatment regimen.

At a median follow-up time of 15.9 months, Abecma led to a statistically significant progression-free survival (PFS) of 13.3 months vs. 4.4 months (p<0.0001), representing a 51% reduction in the risk of disease progression or death. A significant improvement in overall response was also seen (71% overall response, 39% complete response) vs. 42% overall response in the standard treatment group. In those patients who had a complete response or better, median duration of response was 20 months.

In addition, common, serious and fatal side effects can occur with Abecma. In studies, cytokine release syndrome (CRS) of any grade occurred in 89% of patients, neurologic toxicity of any garade in 40% of patients. Other serious and possibly fatal toxicities are outlined in the Boxed Warning in the product label.

Call your healthcare provider or get emergency help right away if you get any of the following side effects:

• difficulty breathing
• fever (100.4°F/38°C or higher)
• chills/shivering
• confusion
• dizziness or lightheadedness
• shaking or twitching (tremor)
• fast or irregular heartbeat
• severe fatigue
• severe nausea, vomiting, diarrhea

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What other treatments have been recently approved to treat multiple myeloma?

Recent treatments include: Elrexfio (2023), Talvey (2023), Carvykti (2022), Tecvayli (2022), Abecma (2021), and Blenrep (2020). Most of the these treatments are used in advanced multiple myeloma, after at least 4 other treatments did not work or have stopped working.

Elrexfio (elranatamab-bcmm) from Pfizer, approved on August 14, 2023.

• Elrexfio is a B-cell maturation antigen (BCMA) CD3-targeted bispecific antibody (BsAb).
• Elrexfio is given as a weekly subcutaneous (under the skin) injection, after two step-up doses, until 24 weeks. At 24 weeks, treatment is then given every two weeks in patients who have achieved at least a partial resoonse and maintained it for at least 2 months.

Talvey (talquetamab-tgvs) from Janssen Biotech, approved on August 9, 2023.

• Talvey is a bispecific GPRC5D-directed CD3 T-cell engaging antibody that binds to the CD3 receptor expressed on the surface of T-cells. This helps to direct the immune system's T cells to fight cancer cells.
• It is given as a subcutaneous (under the skin) injection once every week or once every 2 weeks, after initial step-up dosing in the first week.

Carvykti (ciltacabtagene autoleucel), from Janssen Biotech, approved on February 28, 2022.

• Like Abecma, Carvykti is also a B-cell maturation antigen (BCMA) chimeric antigen receptor (CAR) T cell immunotherapy.
• It is given as a one-time CART-T cell infusion (into a vein in your arm) in a hospital. You will need to remain close to the treatment facility for about one month for monitoring and follow-up from your healthcare providers.

Tecvayli from Janssen, approved on October 25, 2022.

• Tecvayli is a bispecific T-cell engaging antibody. It works by binding to certain areas on cancer cells (CD3 receptor found on the surface of T-cells, and B-cell maturation antigen (BCMA) found on myeloma cells) to help the immune system recognize and kill the cancer.
• It is given as a subcutaneous (under the skin) injection once every week after initial step-up dosing in the first week. After 6 months of successful therapy, some patients may be able to transition to an every 2 week dosing schedule.

Blenrep from GSK, approved August 5, 2020.

• Blenrep is an antibody targeting B-cell maturation antigen (BCMA).
• It is given as an intravenous (IV) infusion (into a vein in your arm) over approximately 30 minutes once every 3 weeks. Your treatment typically continues until the disease progresses or you have unacceptable side effects.

This is not all the information you need to know about Abecma or multiple myeloma for safe and effective use and does not take the place of your doctor’s directions. Review the full product information and discuss this information and any questions you have with your doctor or other health care provider.