Celgene fulfills FDA accelerated approval requirements for Pomalyst/Imnovid (pomalidomide)for multiple myeloma.

Celgene Corporation has announced it has fulfilled the accelerated approval requirements for Pomalyst (pomalidomide) based on results from MM-003, an international phase III study of Pomalyst plus low-dose dexamethasone versus high-dose dexamethasone in relapsed/refractory multiple myeloma patients. Pomalyst, in combination with dexamethasone is approved for patients with multiple myeloma who have received at least two prior therapies including lenalidomide and a proteasome inhibitor and have demonstrated disease progression on or within 60 days of completion of the last therapy.

In the MM-003 study, median progression-free survival (PFS), the primary endpoint of the study, was significantly longer with Pomalyst plus low-dose dexamethasone (3.6 months) than high-dose dexamethasone (1.8 months: HR 0.45 two-sided 95% CI: 0.35-0.59 p < 0.001). Patients in the Pomalyst plus low-dexamethasone arm had a 55% reduction in the risk of progression or death. The pre-specified, final analysis for overall survival (OS) showed a median OS for the Pomalyst plus low-dose dexamethasone arm of 12.4 months (95% CI: 10.4, 15.3), compared to the high-dose dexamethasone arm of 8 months (95% CI: 6.9, 9.0). This survival benefit was statistically significant (HR 0.70 [two-sided 95% CI: 0.54, 0.92], p=0.009) even though 53% of patients in the high-dose dexamethasone arm had subsequently received Pomalyst. The hazard ratio of 0.70 equated to a 30% reduction in the risk of death for patients receiving Pomalyst plus low-dose dexamethasone. Median PFS and OS were based on the assessment of an Independent Review Adjudication Committee. Pomalyst was initially approved by the FDA in February 2013 under the agency's accelerated approval program based on the phase II study, MM-002.

Comment: Pomalyst (Imnovid in the EU), an oral drug, is seen as the successor to Revlimid (lenalidomide) and treats patients no longer responding to Revlimid.Its current competitor is Kyprolis (carfilzomib) with some seven other drugs in late development.