EMA Recommends Granting a Marketing Authorisation for Elotuzumab

EMA fast-tracks treatment of multiple myeloma for approval in EU

On 28 January 2016, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product elotuzumab (Empliciti), intended for the treatment of multiple myeloma. It is to be used in combination with lenalidomide and dexamethasone for the treatment of patients who have received at least one prior therapy.

Empliciti will be available as 300 mg and 400 mg powder for concentrate for solution for infusion.

The active substance of Empliciti is elotuzumab, an immunostimulatory humanised, IgG1 monoclonal antibody (ATC code: L01XC) that specifically targets the SLAMF7 (signalling lymphocyte activation molecule family member 7) protein, highly expressed on multiple myeloma cells.

Multiple myeloma is generally an incurable disease that leads to bone destruction and kidney failure. In 2012, around 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 CHMP reviewed Empliciti under EMA’s accelerated assessment programme. Accelerated assessment is one of the Agency’s main mechanisms to facilitate earlier access by patients to medicines that fulfill unmet medical needs.

The CHMP’s recommendation is based on a randomised, open-label phase III study evaluating Empliciti 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 recurred after, or did not respond to, previous treatment.

In patients also taking Empliciti, a difference in median progression-free survival was 4.2 months in favour to patients taking only lenalidomide and dexamethasone. In addition, 78.5% of patients taking Empliciti with lenalidomide and dexamethasone had a complete or partial response compared to 65.5% in those only taking lenalidomide and dexamethasone.

The most common adverse reactions (occurring in > 10% of patients) with elotuzumab treatment were infusion related reactions, diarrhoea, herpes zoster, nasopharyngitis, cough, pneumonia, upper respiratory tract infection, lymphopenia and weight decrease.

It is proposed that Empliciti therapy should be initiated and supervised by physicians experienced in the treatment of multiple myeloma.

Because multiple myeloma is rare, Empliciti received an orphan designation from the EMA Committee for Orphan Medicinal Products (COMP) 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 ten 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 applicant received scientific advice from the CHMP pertaining to quality and clinical aspects of the dossier.

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 at its January 2016 meeting is an intermediary step on Empliciti’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, 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.

The applicant for Empliciti is Bristol-Myers Squibb Pharma EEIG.

Following this positive CHMP opinion, the COMP will assess whether the orphan designation should be maintained.