Skip to main content

Is Abecma an orphan drug?

Medically reviewed by Leigh Ann Anderson, PharmD. Last updated on April 8, 2024.

Official Answer by Drugs.com

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.

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

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.

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:

Related questions

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.

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

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

Tecvayli from Janssen, approved on October 25, 2022.

Blenrep from GSK, approved August 5, 2020.

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.

References

Read next

How long does Velcade stay in your body?

You could expect Velcade to stay in your system ranging from 9 days (220 hours) to 44 days (1061 hours) depending on which dose you receive. Continue reading

How effective is Velcade for multiple myeloma?

Velcade has been shown in clinical studies to help people with multiple myeloma live longer. In one study of 682 patients who had never received treatment, those who received Velcade plus melphalan and prednisone (MP) lived longer (56.4 months) than people who received melphalan and prednisone alone (43.1 months). Continue reading

Is Darzalex chemotherapy?

Darzalex is NOT chemotherapy it is a monoclonal antibody that may also be called a targeted treatment. Darzalex works by binding to a specific protein called CD38 on the surface of multiple myeloma cells, causing cell death. Continue reading

See also:

Related medical questions

Drug information

Related support groups