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

Updated: Mar 06, 2020
  • Author: Dhaval Shah, MD; Chief Editor: Emmanuel C Besa, MD  more...
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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. [85]

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]). [86]

Also 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. [87]

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