On 21 May 2015, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product pembrolizumab (Keytruda), intended as monotherapy for the treatment of advanced (unresectable or metastatic) melanoma in adults.
The applicant for this medicinal product is Merck Sharp & Dohme Limited.
Pembrolizumab will be available as a 50 mg powder for concentrate for solution for infusion.
The active substance of Keytruda is pembrolizumab, an antineoplastic monoclonal antibody (ATC code L01XC18) that potentiates T cell responses, including anti-tumour responses, through blockade of PD-1 binding to PD-L1 and PD-L2 ligands.
The CHMP based its recommendation on one uncontrolled study and on early results from two ongoing randomised controlled trials (one comparing pembrolizumab with standard chemotherapy and the other comparing pembrolizumab with ipilimumab). The Committee considered that the studies demonstrate the efficacy of pembrolizumab, both in patients who had not previously received ipilimumab and in patients who had previously received ipilimumab.
The benefit of pembrolizumab has been reported in adult patients with advanced (unresectable or metastatic) melanoma who were either ipilimumab naive (overall survival, OS HR=0.69, 95%CI: 0.52, 0.90; p-value < 0.00358; progression-free survival, PFS HR=0.58; 95%CI: 0.47, 0.72; p-value < 0.00001 in comparison to ipilimumab) or previously treated with ipilimumab ((PFS HR=0.57; 95%CI: 0.45, 0.73; p-value < 0.0001 in comparison to chemotherapy).
The most common side effects are diarrhoea, nausea, pruritus, rash, arthralgia and fatigue. Pembrolizumab is associated with immune-related adverse reactions including pneumonitis, colitis, hepatitis, nephritis, endocrinopathies, uveitis, myositis, pancreatitis, and severe skin reactions.
The full indication is: "Keytruda as monotherapy is indicated for the treatment of advanced (unresectable or metastatic) melanoma in adults".
It is proposed that pembrolizumab be prescribed by physicians experienced in the treatment of cancer.
Detailed recommendations for the use of this product will be described in the summary of product characteristics, which will be published in the European public assessment report and made available in all official European Union languages after the marketing authorisation has been granted by the European Commission.
The opinion adopted by the CHMP is an intermediary step on pembrolizumab’s path to patient access. The CHMP opinion will now be sent to the European Commission for the adoption of a decision on an EU-wide marketing authorisation. Once a marketing authorisation has been granted, each Member State will take a decision on price and reimbursement based on the potential role/use of this medicine in the context of its national health system.