Oncopeptides (OTC:ONPPF) said a majority of a panel of the U.S. Food and Drug Administration (FDA) considered that data from a phase 3 trial called OCEAN did not confirm a favorable benefit-risk profile of the company’s blood cancer therapy Pepaxto.
Pepaxto (melphalan flufenamide, also called melflufen) was pulled from the U.S. market by the Swedish company last year in October after a phase 3 trial did not meet criteria of a confirmatory study as per the U.S. FDA. The result also led Oncopeptides to say that it was closing down its commercial business units in the U.S. and Europe.
In February 2021, the FDA granted accelerated approval to Pepaxto, in combination with dexamethasone, to treat adult patients with relapsed or refractory multiple myeloma (RRMM), 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.
The Stockholm based company said in a Sept. 23 press release that the FDA’s Oncologic Drugs Advisory Committee’s (ODAC) were asked the question — “Given the potential detriment in overall survival (OS), failure to demonstrate a progression-free survival (PFS) benefit, and lack of an appropriate dose, is the benefit risk profile of melphalan flufenamide favorable for the currently indicated patient population?” In a 14 to 2 vote, the ODAC answered No.
“We still have confidence in our science and data. The heart of the ODAC discussion was focused on the highly heterogenous survival result in OCEAN across patient groups and how to interpret the subgroup data considering the ITT OS result,” said Oncopeptides CEO Jakob Lindberg.