FDA Approves New Immunotherapy Combination for Relapsed/Refractory Multiple Myeloma

Today, the FDA approved elotuzumab (Empliciti), an injection for intravenous use in combination with pomalidomide and dexamethasone (EPd) for the treatment of adult patients with multiple myeloma who have received at least 2 prior therapies.

The approval was based on data from the ELOQUENT-3 trial, a randomized, open-label, phase 2 study that enrolled 117 patients and found that EPd demonstrated a benefit in patients with relapsed or refractory multiple myeloma. Patients were randomized in a 1:1 ratio to receive either EPd (n = 60) or pomalidomide and dexamethasone (n =57) in 28-day cycles until disease progression or unacceptable toxicity. The study found that the combination treatment doubled both the median progression-free survival and overall response rate versus pomalidomide and dexamethasone alone.

“Empliciti plus pomalidomide and dexamethasone has been proven to extend the time that certain patients live without disease progression, giving healthcare professionals an effective new tool to tackle this relentless cancer,” said Joseph Eid, MD, senior vice president and head of medical, Bristol-Myers Squibb.

The trial specifically demonstrated that the combination treatment reduced the risk of disease progression by 46% (hazard ratio: 0.54; 95% CI: 0.34 to 0.86, P = 0.0078). This finding in turn led to a median progression-free survival of 10.25 months compared with 4.67 months for pomalidomide and dexamethasone alone.

Researchers found that serious adverse events were reported in 22% of the patients treated with EPd, and in 15% of patients treated with pomalidomide and dexamethasone. Treatment discontinuation due to adverse events occurred in 5% of patients enrolled in the EPd arm, compared with 1.8% in the pomalidomide and dexamethasone arm.

“This new regimen of elotuzumab combined with pomalidomide and dexamethasone not only extended the time to disease progression versus a standard of care but also doubled the response rate in some patients whose prior treatments had failed them,” said Paul Richardson, MD, clinical program leader and director of clinical research at the Jerome Lipper Multiple Myeloma Center at Dana-Farber Cancer Institute.

Following priority review by the FDA, EPd is the first triplet combination to be approved on a randomized clinical trial using pomalidomide and dexamethasone as a comparator.