FDA Expands Initial Treatment Options for Multiple Myeloma

Groundbreaking FDA Approvals

The Food and Drug Administration (FDA) recently approved two new treatments that significantly enhance initial therapy options for patients newly diagnosed with multiple myeloma:

1. Daratumumab (Darzalex Faspro): Approved in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for patients eligible for autologous stem cell transplants.

2. Isatuximab (Sarclisa): Approved with VRd for patients ineligible for stem cell transplants.

Both treatments target CD38, a protein frequently found in high amounts on myeloma cells, and integrate into the existing VRd therapy framework to improve patient outcomes.

Key Findings from Clinical Trials

1. Daratumumab (PERSEUS Trial):

• Progression-free survival (PFS) increased significantly (84% in the daratumumab group vs. 68% with VRd alone).

• Higher complete response rates (75% vs. 48%) and sustained minimal residual disease (MRD) negativity (65% vs. 30% after one year).

• Side effects were generally tolerable, with fewer patients discontinuing treatment due to adverse effects (9% vs. 21%).

2. Isatuximab (IMROZ Trial):

• Improved PFS for transplant-ineligible patients (63% in the isatuximab group vs. 45% with VRd alone).

• Sustained MRD negativity for over a year was observed in 47% of isatuximab-treated patients vs. 24% with standard therapy.

• Adverse effects were similar between groups, though higher rates of neutropenia were reported in the isatuximab group.

The Importance of MRD Negativity

MRD negativity, a marker indicating no detectable cancer cells in the bone marrow, is now recognized as a key indicator of treatment success. Patients achieving MRD negativity typically experience longer PFS and overall survival, offering a new metric to evaluate therapy effectiveness.

Unanswered Questions and Ongoing Research

While these advances mark a new era in myeloma treatment, critical questions remain, such as:

• Duration of Maintenance Therapy: Should maintenance therapy continue indefinitely for patients with sustained MRD negativity?

• Optimal Use of CD38-Targeted Therapies: Should these drugs be used upfront, or reserved for second-line treatments?

Ongoing trials, including those sponsored by the National Cancer Institute (NCI), aim to address these questions and refine treatment strategies.

Conclusion

The recent FDA approvals of daratumumab and isatuximab signify a “golden age” for multiple myeloma treatment. With more patients achieving deep responses and prolonged remissions, these therapies represent hope for improving outcomes and quality of life for patients, regardless of their eligibility for stem cell transplants.

Learn More

Explore additional resources on multiple myeloma, treatment advancements, and patient care:

• Plasma Cell Neoplasms (Including Multiple Myeloma)—Patient Version

• Emerging Therapies for Multiple Myeloma

• Understanding MRD Negativity in Cancer Treatment

Stay updated with the latest advancements in oncology at WisePathway.org.