Oncopeptides has decided to withdraw Pepaxto® (melphalan flufenamide) from the US market based on overall survival data from the phase 3 OCEAN study.
In February 2021, Pepaxto in combination with dexamethasone was granted accelerated approval by the FDA to treat adults with relapsed or refractory multiple myeloma who have received at least 4 prior lines of therapy and whose disease is refractory to at least 1 proteasome inhibitor, 1 immunomodulatory agent, and 1 CD38-directed monoclonal antibody.
The randomized, controlled, open-label OCEAN study (ClinicalTrials.gov Identifier: NCT03151811) compared the efficacy and safety of Pepaxto to pomalidomide, in addition to low-dose dexamethasone, in 495 patients with relapsed or refractory multiple myeloma following 2 to 4 lines of prior therapy and who were refractory to lenalidomide in the last line of therapy.
Findings from the study showed an increased risk of death in the Pepaxto treatment arm compared with the pomalidomide arm in the intent-to-treat population, 48% (n=117/246) vs 43% (n=108/249), respectively (hazard ratio 1.104; 95% CI, 0.846-1.441). Median overall survival was 19.7 months (95% CI, 15.1-25.6) for the Pepaxto arm and 25 months (95% CI, 18.1-31.9) for the pomalidomide arm; median follow-up for survival was 19.1 months.
According to the Food and Drug Administration (FDA), data from the OCEAN study were not considered to meet the criteria of a confirmatory study. The Company is working together with the Agency to make the drug available to patients currently receiving Pepaxto.
Oncopeptides withdraws Pepaxto® in US, scale down organization and focus on R&D. News release. Oncopeptides AB. Accessed October 22, 2021. https://www.prnewswire.com/news-releases/oncopeptides-withdraws-pepaxto-in-us-scale-down-organization-and-focus-on-rd-301406644.html.