The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by National Health Service (NHS) in Scotland, has amended its initial decision and now recommends Janssen’s Darzalex (daratumumab) for patients with relapsed or refractory multiple myeloma.
The decision was made after the pharmaceutical company offered to cut the therapy’s cost in Scotland through a patient access program.
The SMC recommends Darzalex as a fourth-line monotherapy treatment option for multiple myeloma patients who were previously treated with at least three prior treatment regimens, but eventually relapsed. Prior treatments must have included a proteasome inhibitor like Velcade (bortezomib) or Kyprolis (carfilzomib), and an immunomodulatory agent.
The consortium noted that the drug would only be available to patients with clear signs of disease progression even after being treated with other approved therapies.
SMC members were sensitive to the arguments of doctors and patient groups that make up the SMC’s Patient and Clinician Engagement (PACE) process, who noted that patients with multiple myeloma ultimately develop resistance to most therapies, and there are limited treatment options available to them.
“Daratumumab offers a further treatment option for those patients with multiple myeloma who have already had a number of previous treatments. Through our PACE meeting, we know this decision will be welcomed by both patients and their families,” SMC chairman Dr. Alan MacDonald said in a news release.
“Janssen welcomes the SMC’s positive recommendation for daratumumab as a monotherapy for relapsed or refractory multiple myeloma,” Jennifer Lee, director of Health Economics/Market Access/Reimbursement (HEMAR) and advocacy at Janssen, said in another press release.
“The rare and aggressive nature of multiple myeloma and the lack of effective and well-tolerated treatment options for patients who have been previously treated and become resistant to other therapies means that there is a real and urgent need for new options like daratumumab,” Lee said.
Darzalex is a monoclonal antibody that works by attaching itself to tumor cells in multiple myeloma patients, triggering their death directly or by allowing the immune system to destroy them. The drug binds to a surface protein called CD38 that exists in certain types of white blood cells, but is most abundant in multiple myeloma cells.
Janssen hopes the National Institute for Health and Care Excellence (NICE), which provides national guidance and advice to improve health and social care in the U.K., follows the SMC and approves the wider use of Darzalex in England and Wales.
Darzalex was approved in the U.S. for the same indication in November 2015. The U.S. approval was the first time Darzalex had been approved in any country, and was based on data from the pivotal open-label Phase 2 MMY2002 trial (NCT01985126).