Celgene (now a subsidiary of Bristol Myers Squibb) received accelerated FDA approval in 2011 for Istodax (romidepsin), a histone deacetylase (HDAC) inhibitor, as monotherapy for the treatment of peripheral T-cell lymphoma (PTCL) in adult patients who have received at least one prior therapy.
This accelerated approval was based upon results from two studies, assessing the effect of Istodax on the surrogate endpoint of overall response rate.
BMS then 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, BM has decided to withdraw the PTCL indication from the US market in accordance with FDA requirements for evaluating accelerated approvals that have not demonstrated sufficient clinical benefit.
Istodax remains on the market for treatment of patients with cutaneous T-cell lymphoma (CTCL) who have received at least one prior systemic therapy.
“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, senior vice president, haematology development at BMS.
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 haematological conditions.