FDA Approval: Belinostat for the Treatment of Patients with Relapsed or Refractory Peripheral T-cell Lymphoma

Cet article présente les données ayant servi de base à l'autorisation de mise sur le marché du belinostat, un inhibiteur d'histone désacétylase, délivrée en juillet 2014 par l'autorité réglementaire américaine pour le traitement des patients atteints d'un lymphome T périphérique récidivant ou réfractaire

Résumé en anglais

On July 3, 2014, the FDA granted accelerated approval for belinostat (BELEODAQ®; Spectrum Pharmaceuticals, Inc.), a histone deacetylase inhibitor, for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). A single-arm, open-label, multicenter, international trial in the indicated patient population was submitted in support of the application. Belinostat was administered intravenously at a dose of 1000 mg/m2 over 30 minutes once daily on Days 1-5 of a 21-day cycle. The primary efficacy endpoint was overall response rate (ORR) based on central radiology readings by an independent review committee (IRC). The ORR was 25.8% (95% CI, 18.3-34.6) in 120 patients that had confirmed diagnoses of PTCL by the Central Pathology Review Group (CPRG). The complete (CR) and partial response (PR) rates were 10.8% (95% CI, 5.9-17.8) and 15.0% (95% CI, 9.1-22.7), respectively. The median duration of response (DoR), the key secondary efficacy endpoint, was 8.4 months (95% CI, 4.5-29.4). The most common adverse reactions (>25%) were nausea, fatigue, pyrexia, anemia, and vomiting. Grade 3/4 toxicities (≥5.0%) included anemia, thrombocytopenia, dyspnea, neutropenia, fatigue, and pneumonia. Belinostat is the third drug to receive accelerated approval for the treatment of relapsed or refractory PTCL.