The Food and Drug Administration (FDA) announced the approval of two BCMA-directed multiple myeloma CAR T-cell therapies for use by relapsed and refractory patients. The approvals signal a major step forward for myeloma patients who need access to these important treatments. 

Before the approval, patients who had relapsed after receiving proteasome inhibitors, immunomodulators, and anti-CD38 monoclonal antibodies (many receiving these as part of their first myeloma treatment) had to wait until relapsing four total lines of therapy before qualifying to receive CAR T therapy. 

These patients struggle to find additional effective treatments and need new options. Data shows that once patients have relapsed after these three key drug classes, the average time in remission is only 3-5 months when other “standard of care” treatments are used. 

It is very exciting for patients to have earlier access to CAR T-cell therapy.

Abecma Approval

On April 4th, 2024, Abecma (ide-cel) by Bristol Myers Squibb was approved by the FDA for patients who had received at least two prior lines of therapy and had been exposed to a proteasome inhibitor, an immunomodulator, and an anti-CD38 monoclonal antibody. 

The approval was based on the KarMMa-3 clinical study, which showed a 51% reduction in the risk of disease progression and a well-established safety profile. In the study, Abecma showed three times the improvement in progression-free survival (or time in remission) compared to the standard of care (13.3 months vs. 4.4 months). 

Frequent side effects for Abecma include cytokine release syndrome (CRS) and neurotoxicity, which were primarily low-grade, and no cases of Parkinsonism were reported. 

“The results of the KarMMa-3 study are remarkable, especially given the historic outcomes with standard regimens for these patients with relapsed or refractory disease,” said Al-Ola A. Abdallah, M.D., University of Kansas, Clinical Associate Professor, Clinical Director, Hematologic Malignancies and Cellular Therapeutics and chair of U.S. Myeloma Innovations Research Collaborative. “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.”

Click here for more information about Abecma. Bristol Myers Squibb notes a 94% manufacturing success rate and has opened additional locations to provide greater access to the treatment. To find locations, click here. 

Carvykti Approval

On April 5th, 2024, Johnson & Johnson's Carvykti (cilta-cel) was approved by the FDA for relapsed/refractory myeloma patients who have received one prior line of therapy, including a proteasome inhibitor and an immunomodulatory drug and are refractory to lenalidomide.

The approval was based on the Phase III CARTITUDE-4 study, which showed that Carvykti reduced the risk of disease progression or death by 59% compared to standard therapies. 

“CARVYKTI demonstrated remarkable efficacy as a personalized, one-time infusion in the earlier treatment of relapsed/refractory multiple myeloma as shown through the CARTITUDE-4 study results,” said Binod Dhakal, M.D., Associate Professor, Medical College of Wisconsin, Division of Hematology and Oncology. “With this approval, I’m excited for patients who may have the opportunity for a treatment-free period for their multiple myeloma as early as first relapse, with the hope of eliminating the burden of having to be on continuous treatment while living with this challenging disease.”

Carvykti, as a potential second-line therapy, could be key in extending the survival of myeloma patients. It is important that these new therapies are available at earlier stages of the disease because only an estimated 15% of myeloma patients are still alive to receive a fifth-line treatment.  

With this very effective treatment, Johnson & Johnson has focused on advancing their manufacturing to scale up Carvykti's production. According to Tyrone Brewer, President of US Hematology, J&J has more than doubled manufacturing of Carvykti in 2023 and is striving to double again in 2024. 

“We will continue to invest in our capacity so we can provide this critical therapy to as many patients as possible.” 

Click here to understand Carvykti’s side effect profile, which is included in the drug packaging. 

Both Abecma and Carvykti offer patients a treatment-free interval, which is a blessing for myeloma patients, who can experience greater freedom from treatment. 

We are grateful for the FDA's willingness to advance myeloma therapies for patients who still need a cure. 

Sources:

• U.S. FDA Approves Bristol Myers Squibb and 2seventy bio’s Abecma for Triple-Class Exposed Relapsed or Refractory Multiple Myeloma After Two Prior Lines of Therapy 
• CARVYKTI® is the First and Only BCMA-Targeted Treatment Approved by the U.S. FDA for Patients with Relapsed or Refractory Multiple Myeloma Who Have Received at Least One Prior Line of Therapy
• Frontline treatment patterns and attrition rates by subsequent lines of therapy in patients with newly diagnosed multiple myeloma