FDA Issues Pepaxto Warning to Multiple Myeloma Patients After Increased Mortality Rate in Trial Participants; What You Need to Know

This move comes just a few months after Pepaxto (melphalan flufenamide) received Accelerated Approval from the FDA.

SurvivorNet obtained a summary of the trial that led to the FDA warning.

Of the 495 individuals tracked in the OCEAN trial, 246 were taking Pepaxto and 249 were taking the controlled combo of pomalidomide and low dose dexamethasone. There were 117 deaths among the Pepaxto users as opposed to 107 for the controlled group, which translates to a mortality rate of 48% versus 43% in the trial.

The median overall survival was also just 19.7 months in the Pepaxto group as compared to 25 months in the control group.

Related: FDA Approves Combination of Keytruda & Lenvima For Treatment Of Advanced Uterine Cancer: What Women Need To Know

Both groups had a large number of deaths due to the high mortality rate among those who Pepaxto aims to treat: adult patients with relapsed or refractory multiple myeloma (cancer that has returned or did not respond to treatment) who have received at least four prior lines of therapy and whose disease was refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody.

Any participants from the trial who believe they are receiving some clinical benefit from Pepaxto may continue treatment, according to the FDA in the OCEAN trial, provided they are informed of the risks and sign a revised written informed consent.

The drug is not yet being recalled, however, and continues to be marketed by the manufacturer, with the FDA noting that it “continues to evaluate the OCEAN trial results and may hold a future public meeting to discuss these safety findings and explore the continued marketing of Pepaxto.”

Patients and health care professionals will be notified when the FDA has any additional information or updates on the drug.

“Patient safety is paramount to Oncopeptides,” the manufacturer said in a statement to SurvivorNet. “The Company has an ongoing dialogue with the FDA and will provide updated information as soon as more information becomes available.”

Oncopeptides is also planning to submit the full data set from the trial to the International Myeloma Workshop meeting in Vienna this September.

Related: Lung Cancer Patients Who Quit Smoking After Diagnosis Live Longer, Slow Spread of Disease, According to New Research

Symptoms of Multiple Myeloma

“It’s very common for someone to say, ‘You know, I’ve been feeling very tired,” or ‘You know, I notice I had a few more infections last winter than I remember,'” said Dr. Shah. “But at the first sign of something abnormal, it’s important to then take that to the next step and say, ‘OK, what am I missing? Is there something else maybe that could be causing this?’ And that’s often when we see a diagnosis of multiple myeloma.”

Bone problems, like pain or weakness in the back, hips, or skull are also symptoms to look out for with multiple myeloma.

After a diagnosis, Dr. Shah said the first step would be to have your blood checked for a level of protein as well as the blood count. There are several other tests doctors will likely perform, including a DNA test. “The DNA is so important because it helps to put people in categories of risk. Once we know that, we’re able to understand how severe the disease is and what we can do to treat it,” explained Dr. Shah.

Learn more about SurvivorNet's rigorous medical review process.

---