Danyelza Approved to Treat Neuroblastoma

November 25, 2020 – The U.S. FDA has approved Danyelza® (naxitamab-gqgk), manufactured by Y- mAbs Therapeutics, for use in combination with granulocyte-macrophage colony-stimulating factor (GMCSF) immunotherapy to treat patients who are at least one year of age, have relapsed or refractory high-risk neuroblastoma in the bone or bone marrow, and have demonstrated a partial response, minor response, or stable disease to prior therapy.

Neuroblastoma is a solid tumor that forms in the nervous system outside of the brain, typically during childhood. While some cases are easily treated, the disease can be extremely aggressive. Patients who have reached stage 4 of the disease are considered high risk, as the cancer has metastasized to other parts of the body, such as the bone and bone marrow. Patients who have multiple copies of the MYCN oncogene, a mutation associated with the development of cancerous cells, are also considered high risk.

In a clinical study, Danyelza used in combination with GMCSF delivered an overall response rate (ORR) of 45%, with 36% of patients having a complete response (CR) and 9% having a partial response (PR). The median duration of response (DOR) was 6.9 months. Thirty percent of responders experienced a DOR of more than six months. A second study of Danyelza used in combination with GMCSF showed an ORR of 34% (26% CR, 8% PR). Twenty-three percent of responders in this study had at least a six-month DOR.

Recommended dosing based on the patient’s weight. Danyelza is administered as an intravenous infusion in an outpatient setting on Days 1, 3, and 5 of each treatment cycle. Treatment cycles are repeated every four weeks until the patient experiences a complete response or partial response, followed by five additional cycles every four weeks. Subsequent cycles may be repeated every eight weeks. Treatment should continue until disease progression or unacceptable toxicity.

A black box warning cautions that there is a risk of serious infusion reactions associated with Danyelza, as well as severe neurotoxicity, including severe neuropathic pain, transverse myelitis, and reversible posterior leukoencephalopathy syndrome (a disorder that affects the cerebral white matter of the brain).

The FDA approved Danyelza under its accelerated approval pathway, meaning that continued approval may be contingent upon verification and description of clinical benefit in confirmatory trials. The drug has been launched at a wholesale acquisition cost (WAC) of $20,368 per vial.