FDA Approves Novartis’ Lutathera for Children with Rare Neuroendocrine Tumors

Rare Daily Staff   

The U.S. Food and Drug Administration approved Novartis’ radioligand therapy Lutathera for the treatment of pediatric patients 12 years and older with rare neuroendocrine tumors.

Lutathera was approved for the treatment of somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut NETs. This approval makes Lutathera the first therapy specifically reviewed and approved for use in pediatric patients with GEP-NETs. Lutathera is also approved in Europe for unresectable or metastatic, progressive, well-differentiated, SSTR-positive GEP-NETs in adults, and in Japan for SSTR-positive NETs.

NETs are a type of cancer that originates in neuroendocrine cells throughout the body and are commonly considered slow-growing malignancies. The diagnosis of NETs is often delayed due to the inactive nature of the disease, and approximately 10 to 20 percent of pediatric patients are diagnosed with metastatic disease. Even though NETs are an orphan disease, their incidence has increased over the past several decades.

“While GEP-NETs in children and adolescents are rare, the impact can be devastating. Today’s approval addresses a critical need for new treatment options for these vulnerable patients,” said Theodore Laetsch, trial investigator and director, Developmental Therapeutics Program, Children’s Hospital of Philadelphia, a NETTER-P clinical trial site. “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.”

The approval was based on the NETTER-P trial, which evaluated Lutathera in patients aged 12 to less than18 years old with SSTR+ GEP-NETs. The study reported a safety profile consistent with the adult population studied in NETTER-1, the pivotal trial for approval of Lutathera in adults. In addition, the estimated radiation absorbed dose in pediatric patients was within established organ thresholds for external beam radiation and comparable to that in adults for the approved dose.

Novartis sees radioligand therapies (RLTs) as having potential to shape the future of cancer care. The company is investigating a portfolio of RLTs to treat a broad range of cancers, including GEP-NETs, lung, prostate, breast, colon, brain, and pancreatic cancers.

Photo: Theodore Laetsch, trial investigator and director, Developmental Therapeutics Program, Children’s Hospital of Philadelphia, a NETTER-P clinical trial site