Danyelza Added to NCCN Guidelines for High-Risk Neuroblastoma

Danyelza is now recommended by the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology as a NCCN category 2a treatment option for patients with high-risk neuroblastoma, a type of brain cancer, according to a news release from Y-mAbs Therapeutics.

“We are very pleased with NCCN’s update of the NCCN Guidelines to include [Danyelza]. We believe this decision reinforces the importance of [Danyelza] as a leading anti-GD2 therapy of choice for physicians treating patients with relapsed/refractory high-risk neuroblastoma,” Doug Gentilcore, SVP, Danyelza business unit head, said in the news release.

Patients in two clinical trials experienced meaningful responses that led to approval of the treatment, with tumors shrinking in the bone, bone marrow or both. In a trial entitled Study 201, 45% of 22 patients saw their cancer shrink, and 30% maintained this response for at least six months. In another trial, Study 12-230, 34% of 38 patients responded; nearly a quarter of patients maintained benefit for at least six months.

Danyelza received accelerated Food and Drug Administration approval on Nov. 25, 2020, for use with granulocyte-macrophage colony-stimulating factor (GM-CSF) in certain patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who had a partial response, minor response or stable disease after prior treatment. Continued approval may depend on results from a confirmatory trial. Danyelza is not approved for osteosarcoma in any country. The approval was based on results from two single-arm trials in this population: Study 201 and Study 12-230.

GM-CSF is a cytokine that helps the body make more white blood cells — especially granulocytes, macrophages and cells that develop into platelets — used as a blood-forming agent in supportive cancer care.

In the two trials, the most commonly reported side effects associated with Danyelza — occurring in at least 25% of patients in either trial — included infusion-related reactions, pain, tachycardia (rapid heartbeat), vomiting, cough, nausea, diarrhea, decreased appetite, hypertension (high blood pressure), fatigue, erythema multiforme (a type of skin reaction), peripheral neuropathy, urticaria (hives), fever, headache, injection site reactions, edema (swelling), anxiety, localized swelling and irritability.

Grade 3 (severe) or 4 (life-threatening) laboratory abnormalities occurring in more than 5% of patients included reductions in lymphocytes, neutrophils, hemoglobin, platelet count, potassium, glucose, calcium, albumin, sodium and phosphate levels, along with elevated alanine aminotransferase (a liver enzyme).

The prescribing information for Danyelza includes a boxed warning about the risk of serious infusion-related reactions and neurotoxicity, such as severe neuropathic pain, transverse myelitis and reversible posterior leukoencephalopathy syndrome.

The recommended regimen includes Danyelza at 2.25 milligrams per kilogram on days 2, 4, 9 and 11, combined with Temodar (temozolomide; 150 milligrams per square meter, days 1 to 5), Camptosar (irinotecan; 50 milligrams per square meter, days 1 to 5) and Leukine (sargramostim; 250 micrograms per square meter, days 6 to 10) in each 28-day cycle. An alternative regimen, based on Unituxin (dinutuximab), uses the same chemotherapy components and also carries a category 2a recommendation.

NCCN, a not-for-profit alliance of 33 leading cancer centers, is committed to improving the quality, effectiveness, equity and accessibility of cancer care and prevention through patient care, research and education. The organization does not guarantee the content it provides and disclaims responsibility for how it is used or applied.

Neuroblastoma is a cancer that starts in immature nerve cells, often in the adrenal glands, abdomen, chest or near the spine. It most commonly affects children under five years old and is usually diagnosed as the tumor grows, causing symptoms.

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