FDA fast-tracks novel MAB neuroblastoma treatment

The monoclonal antibody medicine treats neuroblastoma in the bone and bone marrow.

The FDA recently approved a monoclonal antibody treatment for both pediatric and adult patients to treat neuroblastoma in bone marrow.

Naxitamab-gqgk (Danyelza, Y-mAbs Therapeutics) 40mg/10ml is indicated, in combination with granulocyte-macrophage colony-stimulating factor (GM-CSF), as a treatment treat pediatric 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.

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FDA cleared Danyelza under Priority Review, Orphan Drug, Breakthrough Therapy, and Rare Pediatric Disease designations. Y-mAbs said in a press release that the drug would be available in the “coming weeks”.

Danyelza is a humanized, monoclonal antibody that targets the ganglioside GD2, which is highly expressed in various neuroectoderm-derived tumors and sarcomas. It is administered to patients 3 times in a week in an outpatient setting.

“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,” said Y-mAbs Therapeutics CEO Claus Moller.

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FDA is requiring a boxed warning for Danyelza, noting that it has been shown to cause serious infusion reactions including anaphylaxis, cardiac arrest, bronchospasm, stridor, and hypotension. In addition, the medicine can cause severe pain and hypertension.

The agency’s approval is supported by clinical evidence from 2 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,” YmAbs said.

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