Which medications are FDA approved for treatment of refractory or relapsed multiple myeloma (MM)?

Updated: Jul 15, 2019
  • Author: Dhaval Shah, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Answer

Panobinostat (Farydak) is a histone deacetylase (HDAc) inhibitor approved in February 2015. It is indicated in combination with bortezomib and dexamethasone for treatment of MM in patients who have received at least two prior regimens, including bortezomib and an immunomodulatory agent. The FDA approval was based on efficacy and safety data in a prespecified subgroup analysis of the phase III PANORAMA-1 (PANobinostat ORAl in Multiple MyelomA) trial, in which patients treated with panobinostat (n = 94) had a median progression-free survival of 10.6 months, compared with 5.8 months for patients in the placebo arm (n= 99) (hazard ratio= 0.52 [95% confidence interval: 0.36, 0.76]). [81]

In 2012, the FDA approved carfilzomib (Kyprolis) for the treatment of patients with MM who have received at least two prior therapies including bortezomib and an immunomodulatory agent, and have demonstrated disease progression on or within 60 days of therapy completion. The approval was based on a phase 2b, single-arm, multicenter clinical study of 266 patients with relapsed multiple myeloma with other therapies. The study assessed for overall response rate (ORR), which was 22.9% over a median duration of 7.8 months. [82]

In 2015, the FDA expanded carfilzomib’s indication for multiple myeloma based on data from the ASPIRE study. In this study, carfilzomib was combined with lenalidomide and dexamethasone (KRd) for patients with relapsed multiple myeloma who had received 1-3 prior lines of therapy. The study showed a significant improvement in progression-free survival (PFS) for patients treated in the KRd arm compared with those treated with lenalidomide and low-dose dexamethasone (Rd) alone. The median PFS was 26.3 months in the KRd arm compared to 17.6 months in the Rd arm. [83]

In January 2016, the FDA approved carfilzomib in combination with dexamethasone for relapsed or refractory multiple myeloma in patients who have received 1-3 prior lines of therapy. Approval was based on the ENDEAVOR study (n=929) where a statistically significant improvement in median progression-free survival was observed with carfilzomib plus dexamethasone compared with bortezomib plus dexamethasone in patients with relapsed multiple myeloma (26.3 mo vs 17.6 mo; p=0.0001). Overall survival data are not yet available. [84]


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