The therapy had received accelerated approval in 2011 but a phase 3 confirmatory trial found it did not meet the end point of progression-free survival.
Bristol Myers Squibb has withdrawn the indication for Istodax (romidepsin) as a monotherapy for the treatment of peripheral T-cell lymphoma (PTCL) in adult patients who have received at least one prior therapy. In 2011, Celgene, now a subsidiary of BMS, received accelerated approval for this indication. The approval was based on results from two clinical studies that assessed the effect of Istodax on the surrogate end point of overall response rate.
Bristol Myers Squibb conducted a confirmatory phase 3 study evaluating Istodax plus a combination regimen of cyclophosphamide, doxorubicin, vincristine, and prednisone (CHOP) versus just CHOP as a first-line treatment of PTCL patients, but results from the trial showed that Istodax did not meet the primary efficacy end point of progression-free survival.
Istodax remains on the market for treatment of patients with cutaneous T-cell lymphoma (CTCL) who have received at least one prior systemic therapy. Patients who are being treated with Istodax for PTCL may remain on treatment if doing so ius deemed clinically appropriate by the treating physician.
“While the outcome of the confirmatory study in peripheral T-cell lymphoma is disappointing, Bristol Myers Squibb will continue to provide Istodax for patients with cutaneous T-cell lymphoma, where it remains an approved and important treatment option,” Noah Berkowitz, M.D., Ph.D., senior vice president, hematology development at Bristol Myers Squibb, said in a statement issued yesterday. “As always, our efforts across blood cancer research and development remain centered on delivering better outcomes for patients in need.”
This is not the first time an indication has been withdrawn for a BMS oncology therapy that was approved on an accelerated basis after the drug fell short in a confirmatory trial. In July, the company announced it was withdrawing the hepatocellular carcinoma indication for Opdivo (nivolumab), its groundbreaking PD-1 inhibitor. Opdivo had received accelerated approval in 2017, making it the first immunotherapy to be approved for this use.
The approval was based on tumor responses from the phase 1/2 CheckMate-040 trial. In the confirmatory trial, CheckMate-459, Opdivo was compared with Nexavar (sorafenib) in the first-line setting. The results showed that Opdivo did not achieve statistical significance for its primary end point of overall survival.