FDA Grants Breakthrough Therapy Designation to Daratumumab for Multiple Myeloma

The US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for injected Darzalex (daratumumab) added to standard-of-care treatment regimens among patients with relapsed or refractory multiple myeloma, Genmab A/S has announced.

The FDA designation applies to daratumumab in combination with dexamethasone and the immunomodulatory agent lenalidomide (Revlimid), or dexamethasone and the proteasome inhibitor bortezomib (Velcade), among patients who have undergone at least one prior line of therapy.

Under the 2012 FDA Safety and Innovation Act, Breakthrough Therapy Designation allows accelerated regulatory review  of potential new treatments for life-threatening diseases when early clinical evidence suggests substantial improvement over existing treatments. The agency granted this designation to daratumumab based on data from the ongoing phase III CASTOR (NCT02136134) and POLLUX (NCT02076009) clinical trials. CASTOR compares bortezomib and dexamethasone with and without daratumumab; POLLUX is evaluating lenalidomide and dexamethasone with and without daratumumab.

Daratumumab is an intravenously-infused human IgG1k monoclonal antibody (mAb) with high binding affinity to CD38, which is expressed on multiple myeloma cell surfaces. It is the first monoclonal antibody that has been approved by the FDA for treating multiple myeloma. In 2015, the FDA granted it accelerated approval for the treatment of patients with multiple myeloma who have received three or more prior lines of therapy, including a proteasome inhibitor and an immunomodulatory agent, or who are refractory to both.

Daratumumab is undergoing development by Janssen Biotech under an exclusive worldwide license from Genmab. Janssen is conducting several randomized studies of daratumumab for multiple myeloma, using progression-free survival time as a primary trial endpoint.