The European Medicines Agency (EMA) has fast-tracked the approval of the drug elotuzumab (Empliciti) for the treatment of multiple myeloma.
The drug helps to activate the body’s immune system to fight against the cancer, and is to be used in combination with lenalidomide and the anti-inflammatory medicine dexamethasone for the treatment of patients who have received at least one prior therapy.
Elotuzumab is a monoclonal antibody that works by activating the body’s immune system to attack and kill multiple myeloma cells.
Multiple myeloma is a rare malignant disease of a type of white blood cells called plasma cells. In multiple myeloma, the division of plasma cells becomes out of control, resulting in abnormal, immature plasma cells multiplying and filling up the bone marrow. The abnormal cells interfere with the production of normal white blood cells, red blood cells and platelets, and patients develop complications such as infections, anaemia, bone pain and fractures, raised blood calcium levels and kidney dysfunction.
Multiple myeloma is generally an incurable disease that leads to bone destruction and kidney failure. In 2012, about 39,000 people had multiple myeloma in the European Union (EU). Only half of patients diagnosed with multiple myeloma are still alive after five years under currently available treatment. Therefore new medicines are needed for patients whose disease returns after treatment.
The Committee for Medicinal Products for Human Use (CHMP) reviewed elotuzumab under the EMA’s accelerated assessment programme. Accelerated assessment is one of the Agency’s main mechanisms to facilitate earlier access by patients to medicines that fulfil unmet medical needs.
The CHMP’s recommendation is based on a randomised, open-label Phase 3 study evaluating elotuzumab in combination with lenalidomide and dexamethasone versus lenalidomide and dexamethasone alone in patients with relapsed or refractory multiple myeloma. The trial enrolled 646 patients whose multiple myeloma came back after, or did not respond to, previous treatment. In patients also taking elotuzumab, the disease progressed more slowly (difference in medians of 4.2 months) than in patients taking only lenalidomide and dexamethasone.
In addition, 78.5% of patients taking elotuzumab with lenalidomide and dexamethasone saw a complete or partial shrinkage of their tumours compared to 65.5% in those only taking lenalidomide and dexamethasone.
The most common side effects of elotuzumab are infusion-related reactions, diarrhoea, cough, shingles, nasopharyngitis (infection of the nose and throat), pneumonia, upper respiratory tract infection, lymphopenia (lowered counts of a type of white blood cell called lymphocytes) and weight loss.
Because multiple myeloma is rare, elotuzumab received an orphan designation from the Committee for Orphan Medicinal Products in 2012. Orphan designation is the key instrument available in the EU to encourage the development of medicines for patients with rare diseases. Orphan-designated medicines qualify for 10 years’ market exclusivity. In addition, orphan designation gives medicine developers access to incentives, such as fee reductions for marketing authorisation applications and scientific advice.
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, decisions about price and reimbursement will take place at the level of each member state, taking into account the potential role/use of this medicine in the context of the national health system of that country.