The FDA’s oncology department has lately been tightening its act around accelerated approvals and stressing the importance of patient survival data. Several companies immediately fell in line, withdrawing problematic indications when the agency mounted its pressure. But some companies have decided to put up a fight.
Secura Bio’s PI3K inhibitor Copiktra and Oncopeptides’ first-in-class blood cancer therapy Pepaxto are the latest two drugs that will be scrutinized at an upcoming FDA advisory committee meeting, as the drugmakers haven’t pulled indications off the market despite red flags from clinical trials.
During a two-day event in September, external experts for the FDA will discuss whether Copiktra’s use in third-line chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) and Pepaxto’s use alongside dexamethasone for fifth-line treatment of multiple myeloma should stay on, according to an FDA filing.
Both drugs have shown concerning signs of potential detriment to patients’ lives in their confirmatory trials, and the FDA has alerted the public of those risks related to Pepaxto and Copiktra.
Last October, Oncopeptides had decided to voluntarily take Pepaxto off the U.S. market but changed its mind this January after conducting additional analyses of patient survival data from the phase 3 OCEAN study. The company has put marketing of Pepaxto on hold “until a mutual understanding” with the FDA.
In the original analysis of OCEAN data, Pepaxto’s combo with dexamethasone was linked to a 10% increased risk of death compared with Bristol Myers Squibb’s Pomalyst and dexamethasone in a group of patients with relapsed or refractory multiple myeloma after two to four lines of prior therapy. The peptide-drug conjugate originally got its accelerated approval last February based on data from the phase 2 HORIZON trial showing Pepaxto triggered a 23.7% response rate in a small group of patients who had received at least four prior lines of therapy.