The results of study MM-003, comparing pomalidomide plus low-dose dexamethasone versus high-dose dexamethasone alone have been released by Celgene International Sari (Boudry, Switzerland). This Phase III, multicenter, randomized, open label trial indicates that the pomalidomide arm of the study shows both improved progression free survival (PFS) and overall survival (OS). Professor Jesus San Miguel, Head of the Department of Hematology at the University of Salamanca in Spain and principal investigator for the study, was clearly very encouraged by the results indicating that: “The survival results in this study build on earlier observations of high response rates for pomalidomide and dexamethasone in multiple myeloma.”
The benefit in patients who relapsed on or were refractory to (having ultimately progressed on) at least 2 prior therapies, including both lenalidomide (Revlimid®) and bortezomib (Velcade®) is impressive and would seem to meet the FDA criteria for filling an “unmet need” for refractory/relapsing myeloma patients. When the FDA recently cancelled the planned ODAC hearing for pomalidomide (see prior blog), there were questions about what that might mean. However, today’s announcement really increases the likelihood of favorable review of pomalidomide by the FDA and potential early approval. The pomalidomide combination has, in addition, been submitted to the EMA (regulatory agency) in Europe with expectation of a decision in 2013. The EMA and the FDA have regular joint conference calls to review joint submissions and share data.
For more information, see Celgene's press release "Pomalidomide Demonstrated Significant Progression-Free Survival and Overall Survival Advantages in Phase III Study of Heavily Pre-Treated Myeloma Patients".