The U.S. Food and Drug Administration (FDA) has approved naxitamab (Danyelza) to treat neuroblastoma in the bone or bone marrow.
Naxitamab, a monoclonal antibody that targets the ganglioside GD2, is approved in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF) for pediatric patients one year of age and older and for adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow demonstrating a partial response, minor response, or stable disease to prior therapy.
Neuroblastoma is a solid tumor that arises in the peripheral nervous system in children. Patients are staged based on the International Neuroblastoma Staging System Committee (INSS) system, ranging from stage 1 through stage 4S. Those with the high-risk, stage 4 disease show neuroblastoma cancer cells that have metastasized to other areas of the body, including the bone or bone marrow.
The approval is largely based on two single-arm, open-label trials [Study 201 (NCT 03363373) and Study 12-230 (NCT 01757626)] showing overall response rates of 45% and 34%, respectively.
The drug comes with a Boxed Warning stating that naxitamab can cause serious infusion-related reactions and neurotoxicity, including severe neuropathic pain, transverse myelitis, and reversible posterior leukoencephalopathy syndrome (RPLS). Patients should receive premedication prior to each naxitamab infusion and be closely monitored during each infusion and for at least two hours following completion.
The recommended dose for naxitamab is 3 mg/kg/day (up to 150 mg/day) on days 1, 3, and 5 of each treatment cycle, administered after dilution as an intravenous infusion in combination with GM-CSF, subcutaneously at 250 µg/m2/day on days -4 to 0 and at 500 µg/m2/day on days 1 to 5. Cycles are repeated every 4 to 8 weeks.
Common adverse reactions for naxitamab were infusion-related reactions, pain, tachycardia, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, injection site reaction, edema, anxiety, localized edema, and irritability.
Since the drug is applicable to pediatric populations, the developer, Y-mAbs Therapeutics, received a priority review voucher that can be used or transferred to another company. Recently, Eiger Biopharmaceuticals sold their review voucher for $95 million which will allow the new owner to guarantee their drug will be reviewed by the FDA in less than 6 months instead of the standard 10 months.
To keep up to date on the latest FDA approvals, sign up for our newsletter at https://checkrare.com/sign-up/