FDA Accelerates Approval of Danyelza for Children and Adults with Relapsed or Refractory High-Risk Neuroblastoma

The Food and Drug Administration (FDA) granted accelerated approval for Danyelza (naxitamab) with granulocyte-macrophage colony-stimulating factor for the treatment of patients with relapsed or refractory high-risk neuroblastoma in the bone marrow or bone who have had a partial or minor response, or shown stable disease to previously administered therapies.

The therapy is both for children one year and older and adults, according to an alert from the FDA. The drug’s efficacy was assessed in two trials: Study 201 and Study 12-230. Both trials included patients with relapsed or refractory neuroblastoma in the bone marrow or bone, and patients received a 3 mg/kg of Danyelza intravenously on days one, three and five of each four-week cycle, in addition to granulocyte-macrophage colony-stimulating factor subcutaneously, or under the skin, at varying doses throughout the cycle. Patients were allowed to receive preplanned radiation in specific areas based on which trial they were enrolled in.

In Study 201, the overall response rate (ORR), the main efficacy outcome, was 45%, with 30% of patients who responded to the therapy having a duration of response lasting for at least six months. Study 12-230 found that patients achieved an ORR of 34% with 23% of patients having a duration of response of at least six months. Responses to the drug in both trials were observed in the bone marrow, bone or both.

A boxed warning was included in the prescribing information, informing patients and clinicians about serious infusion-related reactions and neurotoxicity related to Danyelza. Patients should receive premedication before receiving Danyelza in an attempt to mitigate these risks, in addition to monitoring during the treatment and for at least two hours after completion.

Some common adverse reactions with Danyelza, which occurred in at least 25% of patients from both trials, include pain, infusion-related reactions, vomiting, tachycardia (a type of irregular heartbeat), nausea, cough and headache, among others. Some Grade 3 or 4 abnormalities, which are the more serious kinds, that occurred in at least 5% of patients in both trials include decreased platelet count, decreased calcium and decreased glucose, in addition to others.

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