Novartis’ Radioligand Therapy Gains FDA Nod for Pediatric Patients

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FDA has granted approval for the use of Novartis’ radioligand therapy, Lutathera, to treat pediatric patients with gastroenteropancreatic neuroendocrine tumors.

Novartis announced, in an April 23, 2024 press release, that its radioligand therapy, Lutathera, has gained FDA approval as a treatment for pediatric patients aged 12 years and older with somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs), inclusive of foregut, midgut, and hindgut GEP-NETs.

“Lutathera is now the very first therapy approved specifically for children with GEP-NETs, offering new hope to young patients living with this rare cancer,” said Tina Deignan, Therapeutic Area head, Oncology US, Novartis, in the press release (1). “Radioligand therapies have extraordinary potential to shape the future of cancer care. With this approval, we have taken another vital step toward fulfilling that vision, strengthening our commitment to researching and developing the RLT [radioligand therapy] platform across multiple cancer types and treatment settings.”

FDA’s approval was based on the ongoing open-label, multicenter, single-arm, Phase II NETTER-P trial, which has been designed to evaluate the safety and dosimetry of Lutathera in patients between the ages of 12 and 18 years old who have SSTR+ GEP-NETs (2). The results of the Phase II NETTER-P trial were consistent with those found in adult patients via the NETTER-1 trial.

“While GEP-NETs in children and adolescents are rare, the impact can be devastating. [This] approval addresses a critical need for new treatment options for these vulnerable patients,” said Dr. Theodore Laetsch, trial investigator and director, Developmental Therapeutics Program, Children’s Hospital of Philadelphia (CHOP), a NETTER-P clinical trial site, in the press release (1). “The introduction of radioligand therapy significantly advanced how we treat GEP-NETs, and I’m encouraged that younger patients now have the potential to benefit from this innovation.”


Additionally, in January 2024, Novartis announced the results of the Phase III NETTER-2 trial, demonstrating that Lutathera plus long-acting release (LAR)octreotide reduced the risk of disease progression or death by 72% when used as a first-line therapy in adult patients with SSTR+ well-differentiated grade 2/3 advanced GEP-NETs (3).

“This is the first positive Phase III trial of a radioligand therapy in the first-line setting, and the overall efficacy and safety results are amongst the most clinically relevant observed to date in this kind of advanced cancer, addressing a significant unmet need for patients with newly diagnosed advanced GEP-NETs,” said Jeff Legos, global head of Oncology Development at Novartis, in a press release (3). “The positive results are a significant advancement and further reaffirm our strategy to research and develop radioligand therapies in earlier lines of treatment or stages of disease to improve outcomes for patients.”


1. Novartis. Novartis Radioligand Therapy Lutathera FDA Approved as First Medicine Specifically for Pediatric Patients with Gastroenteropancreatic Neurodocrine Tumors. Press Release, April 23, 2024.
2. NIH. Study to Evaluate Safety and Dosimetry of Lutathera in Adolescent Patients with GEP-NETs and PPGLs. (accessed April 24, 2024).
3. Novartis. Novartis Lutathera Significantly Reduced Risk of Disease Progression or Death by 72% as First-Line Treatment for Patients with Advanced Gastroenteropancreatic Neuroendocrine Tumors. Press Release, Jan. 19, 2024.

Source: Novartis