FDA approves Danyelza for high-risk neuroblastoma
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The FDA granted accelerated approval to naxitamab-gqgk for children aged 1 year or older and adults with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow, according to the drug’s manufacturer.
Naxitamab-gqgk (Danyelza, Y-mAbs Therapuetics) — a humanized, monoclonal antibody that targets the ganglioside GD2 — is indicated for use in combination with granulocyte-macrophage colony-stimulating factor for patients who have achieved partial or minor response or stable disease with prior treatment.
The FDA granted the accelerated approval based on overall response rate and duration of response observed with the therapy in two studies of patients with high-risk, relapsed or refractory neuroblastoma.
Naxitamab-gqgk also appeared well-tolerated, with few patients discontinuing treatment due to adverse events. The most common adverse events associated with treatment 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.
“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,” Claus Moller, CEO of Y-mAbs, said in a company-issued press release. “This approval of Y-mAbs’ first [biologics license application] represents a key step in working toward our mission of becoming a world leader in developing better and safer antibody-based oncology products addressing unmet pediatric and adult medical needs.”
Naxitamab-gqgk will undergo further evaluation in an FDA-required postmarketing study. The ongoing Study 201 plans to enroll at least 80 patients to evaluate ORR as a primary endpoint and duration of response, PFS and OS as secondary endpoints.
This application also received priority review, orphan drug, breakthrough therapy and rare pediatric disease designations from the FDA.
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