The European Medicines Agency's (EMA): Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Pomalidomide in combination with dexamethasone for the treatment of relapsed and refractory multiple myeloma (rrMM) in patients who have received at least two prior therapies, including both lenalidomide and bortezomib, and have demonstrated disease progression while on their last therapy.
Multiple myeloma is the second most common cancer of the blood and accounts for around 1% of all cases of cancer in the UK. Multiple myeloma remains incurable and nearly all patients who achieve an initial response to treatment will relapse and require additional treatment options.
"Celgene is the first company in more than 40 years to deliver two oral treatments for multiple myeloma," said Alan Colowick, MD, President of Celgene Europe, the Middle East and Africa (EMEA). "These treatments continue to play an important role in increasing overall survival for patients living with this rare disease. Following the final decision by the European Commission within the next few months, we hope to bring oral pomalidomide, Celgene's third and important treatment option for patients who have fully benefitted from other treatments including lenalidomide and need new options to help treat their disease. Our commitment to patients with this disease is a centrepiece of who we are as an organisation and our focus on rare and debilitating diseases."
The CHMP positive opinion was based on the results of the MM-003 study, a Phase III, multi-centre, randomised (2:1), open-label study in 455 patients. The trial evaluated use of oral pomalidomide plus low-dose dexamethasone (n=302) compared with high-dose dexamethasone (n=153) in patients with rrMM. According to the protocol, all patients were to have been treated with both bortezomib and lenalidomide prior to study entry. In the trial, progression-free survival, the primary endpoint, was significantly longer in patients who received pomalidomide plus low-dose dexamethasone (15.7 weeks) compared with those who received high-dose dexamethasone alone (8 weeks [HR, 0.45; P<0.001]). Median overall survival, the secondary endpoint, was also significantly improved for pomalidomide plus low-dose dexamethasone arm, compared with high-dose dexamethasone only (due to patients still on therapy in the pomalidomide plus low-dose dexamethasone arm, the median has not yet been reached vs. 34 weeks in the high-dose dexamethasone arm [HR, 0.53; P<0.001]).
The most common grade 3/4 haematologic toxicities for the pomalidomide plus low-dose dexamethasone arm and high-dose dexamethasone arms were neutropenia, thrombocytopenia and febrile neutropenia.3
The European Commission, which generally follows the recommendation of the CHMP, is expected to make its final decision within two to three months.