FDA Approves Y-mAbs’ DANYELZA® (naxitamab-gqgk) for the Treatment of Neuroblastoma
“Today is an important day for children living with refractory/relapsed high-risk neuroblastoma,” said
“We believe that DANYELZA in combination with GM-CSF is a much-needed treatment for patients with relapsed/refractory high-risk neuroblastoma in the bone or bone marrow who have historically not had approved treatments available. This approval of Y-mAbs’ first BLA represents a key step in working towards our mission of becoming a world leader in developing better and safer antibody-based oncology products addressing unmet pediatric and adult medical needs,” said
The FDA approval of DANYELZA is supported by clinical evidence from two pivotal studies in patients with high-risk neuroblastoma with refractory or relapsed disease. DANYELZA appears to be well tolerated with few discontinuations of treatment in the clinical trials and adverse events were clinically manageable. See below for information related to adverse reactions.
The FDA granted approval under the accelerated approval regulation. The postmarketing clinical trial required by the FDA to verify and to further characterize the clinical benefit is the ongoing Study 201, which will enroll a minimum of 80 patients and report overall response rate (“ORR”), duration of response (“DOR”), progression free survival (“PFS”) and overall survival (“OS”). The ORR is the primary endpoint for the study, DOR is the secondary endpoint, PFS and OS are secondary endpoints in long-term follow up.
DANYELZA is expected to be available in
About High-Risk Neuroblastoma
Neuroblastoma is a solid tumor of childhood that arises in the nervous system, outside of the brain. The clinical behavior of neuroblastoma is highly variable, with some tumors being easily treatable, but the majority being very aggressive. All patients are staged based on the International Neuroblastoma Staging System Committee (“INSS”) system, ranging from stage 1 through stage 4S. All patients with stage 4 disease diagnosed after one year of age are classified in the high-risk category, where the neuroblastoma tumor cells have already metastasized to other sites in the body, such as the bone or bone marrow. Essentially all patients who have tumors with many copies, or amplification, of the MYCN oncogene also have high-risk disease, even if they do not have evidence of the tumor having spread.
Important Safety Information and Indication for DANYELZA® (naxitamab-gqgk)
DANYELZA (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefits in a confirmatory trial.
Important Safety Information
Please click here to see the full Prescribing Information for DANYELZA.
DANYELZA is contraindicated in patients with a history of severe hypersensitivity reaction to naxitamab-gqgk. Reactions have included anaphylaxis.
Warnings and Precautions
DANYELZA has been approved with a box warning.
In clinical studies, DANYELZA has been shown to cause serious infusion reactions including anaphylaxis, cardiac arrest, bronchospasm, stridor, and hypotension. Infusion reactions generally occurred within 24 hours of completing a DANYELZA infusion, most often within 30 minutes of initiation. Infusion reactions are most frequent during first infusion in each cycle. Premedicate with an antihistamine, acetaminophen, an H2 antagonist and corticosteroid as recommended in the label. Monitor patients closely for signs and symptoms of infusion reactions during and for at least 2 hours following completion of each DANYELZA infusion in a setting where cardiopulmonary resuscitation medication and equipment are available. Reduce the rate, interrupt infusion, or permanently discontinue DANYELZA based on severity and institute appropriate medical management as needed.
Based on its mechanism of action, DANYELZA can cause severe pain. Premedicate with gabapentin and e.g. oral oxycodone. Treat break-through pain with intravenous hydromorphone or equivalent.
One case of transverse myelitis (Grade 3) has been reported. Permanently discontinue DANYELZA therapy in case of transverse myelitis.
DANYELZA may cause severe hypertension. The onset of hypertension may be delayed. Monitor blood pressure during and after infusion. Interrupt DANYELZA infusion and resume at a reduced rate, or permanently discontinue DANYELZA based on the severity.
Two cases of posterior reversible encephalopathy syndrome (“PRES”) have been reported. Monitor blood pressure during and following DANYELZA infusion and assess for neurologic symptoms. Permanently discontinue DANYELZA in case of symptomatic PRES.
The most common adverse events were mainly mild and moderate and included infusion-related reaction, pain, tachycardia, vomiting, cough, nausea, diarrhea, decreased appetite, hypertension, fatigue, erythema multiforme, peripheral neuropathy, urticaria, pyrexia, headache, edema, anxiety, localized edema and irritability.
This is not the complete list of Warnings, Precautions and Adverse Reactions. For further information see label.
To report suspected adverse reactions, contact
Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer. The Company has a broad and advanced product pipeline, including DANYELZA and omburtamab, which target tumors that express B7-H3.
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