Bristol Myers Squibb has withdrawn the U.S. indication for Istodax for relapsed/refractory peripheral T-cell lymphoma.
Istodax was approved under the accelerated approval pathway, but failed to meet the efficacy endpoint in a confirmatory trial.
A statement by BMS follows:
In 2011, Celgene Corporation, now a wholly owned subsidiary of Bristol Myers Squibb, received accelerated approval by the U.S. Food & Drug Administration for Istodax (romidepsin), a histone deacetylase inhibitor, as monotherapy for the treatment of peripheral T-cell lymphoma in adult patients who have received at least one prior therapy.
This accelerated approval was based upon results from two clinical studies, assessing the effect of Istodax on the surrogate endpoint of overall response rate. Bristol Myers Squibb conducted a subsequent confirmatory phase III study evaluating romidepsin plus CHOP (Ro-CHOP) versus CHOP in first-line PTCL patients, but the trial did not meet the primary efficacy endpoint of progression free survival.
Based on this outcome, Bristol Myers Squibb made the decision to withdraw the PTCL indication from the U.S. market. The company took this action in accordance with the FDA’s requirements for evaluating accelerated approvals that have not demonstrated sufficient clinical benefit.
Bristol Myers Squibb is notifying healthcare professionals about the withdrawal. 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 should consult with their healthcare provider in all aspects of their medical care and may remain on treatment if deemed clinically appropriate by the treating physician. For patients who are currently being treated with Istodax for PTCL, and who have questions about access and reimbursement support, please contact BMS Access Support at 1-800-861-0048 or visit www.bmsaccesssupport.com.
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,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb. “As always, our efforts across blood cancer research and development remain centered on delivering better outcomes for patients in need.
Since the initial approval of Istodax, nearly a decade ago, more options have been made available for patients, many of which have redefined treatment across PTCL and other hematologic conditions.
