NICE rejects use of daratumumab (darzalex) monotherapy for treatment of myeloma

The UK National Institute for Health and Care Excellence (NICE) has issued draft negative guidance on anti-cancer drug daratumumab (darzalex) monotherapy, to be used on its own and not in combination with other drugs.

Also known as multiple myeloma, myeloma is a cancer caused by abnormal cells in the bone marrow, and patients suffering from the condition experience a number of relapses and remissions while the disease becomes resistant to treatment.

With the rejection, daratumumab will not be made available for National Health Service (NHS) patients with relapsed and refractory myeloma.

Myeloma UK Policy and Public Affairs manager Kate Morgan said: "This is a very disappointing decision for myeloma patients in England and Wales, but it was always going to be a challenging appraisal.

"We understand why NICE has not been able to say yes at this point given there are major limitations associated with the early nature of the trial data and the lack of direct comparison with the current standard of care.

"However, since daratumumab was given an accelerated licence by the European licensing body, this negative decision demonstrates the need for NICE and other stakeholders to be better prepared to deal with situations like this."

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells.

"This is a very disappointing decision for myeloma patients in England and Wales, but it was always going to be a challenging appraisal."

It is being developed by Janssen Biotech under an exclusive worldwide licence to develop, manufacture and commercialise daratumumab from Genmab. 

Last month, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending broadening the existing marketing authorisation for daratumumab in the European Union. 

It has received two Breakthrough Therapy Designations from the US FDA, for multiple myeloma, as both a monotherapy and in combination with other therapies. 

Myeloma UK intends to work in collaboration with NICE and Janssen to find out solutions related to critical issues that arise in appraisals based on early data.