Oncopeptides has announced that the US Food and Drug Administration, FDA, has requested a withdrawal of the US marketing authorization for Pepaxto.
The request is based on the outcome of the confirmatory phase 3 OCEAN study, which demonstrated an ITT overall survival HR of 1.1, but with significant survival result differences for both melflufen and the comparator drug pomalidomide for large relevant patient groups.
“We respect FDA’s accelerated approval regulations,” says Jakob Lindberg, CEO of Oncopeptides. “Multiple myeloma remains an incurable disease, and the treatment options for patients with triple class refractory disease will ultimately become exhausted. The OCEAN study demonstrated clinical benefit for multiple myeloma patients, in particular for non-transplanted elderly patients where the unmet medical need remains very high.”
Pepaxto in the US
Pepaxto was granted accelerated approval in the U.S., on February 26, 2021, and is indicated in combination with dexamethasone, for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one CD38-directed monoclonal antibody. At the FDA’s request, Oncopeptides stopped marketing Pepaxto in the US on October 22, 2021, and Pepaxto is currently not commercially available for US patients.
Pepaxto in Europe
The commercialization of Pepaxti in Europe is ongoing. Pepaxti has a full approval from the European Medicines Agency, EMA, since August 18, 2022, and was approved by the Medicines and Healthcare Products Regulatory Agency, MHRA, in the UK on November 11, 2022. Both approvals take the large OCEAN study overall survival differences across relevant patient groups into account. Pepaxti is indicated in combination with dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least three prior lines of therapies, whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and one anti-CD38 monoclonal antibody, and who have demonstrated disease progression on or after the last therapy. For patients with a prior autologous stem cell transplantation, the time to progression should be at least 3 years from transplantation.
Photo of Jakob Lindberg: Oncopeptides