FDA Approves Lutathera for Adolescents with Neuroendocrine Tumors

The FDA has approved Novartis’ Lutathera (lutetium Lu 177 dotatate) to treat adolescents 12 years of age and older with somatostatin receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine tumors (GEP-NETs). Lutathera is already approved to treat adults with these tumors.

Neuroendocrine tumors are a type of cancer that originates in neuroendocrine cells throughout the body and are commonly considered slow-growing malignancies. About 10% to 20% of pediatric patients are diagnosed with metastatic disease. More than 12,000 people in the United States are diagnosed with a NET each year, according to Cancer.net.

Lutathera is a radiotherapeutic that was approved in 2018 to treat adults with gastroenteropancreatic neuroendocrine tumors. It belongs to a class called Peptide Receptor Radionuclide Therapy (PRRT), a form of targeted treatment comprised of a targeting molecule that carries a radioactive component. The targeting molecule binds to a specific receptor on tumor cells, and is then internalized into the target cells, where the radioactive component destroys the tumor cells.

The list price (wholesale acquisition cost) of Lutathera is $55,896, and most patients require about four doses, according to a company spokesperson. The dosage is the same for adults and adolescents.

"Novartis aims to price its medicines based on the value they deliver to patients, healthcare systems and society," the spokesperson said. "We have taken a thoughtful approach to the pricing of Lutathera, balancing the innovation it brings to the treatment of GEP-NETs, with the benefits it can provide to patients and the importance of ensuring that appropriate patients have access to it.

Novartis offers a $25 copay program for commercially insured patients up to a maximum annual benefit of $15,000. The terms state: “The value of this Program is exclusively for the benefit of patients and is intended to be credited toward patient out-of-pocket obligations and maximums, including applicable co-payments, coinsurance, and deductible.”

For the full year 2023, Lutathera generated sales of $605 million, up 28% from 2022.

“Today’s approval addresses a critical need for new treatment options for these vulnerable patients,” said Theodore Laetsch, M.D., trial investigator and director, Developmental Therapeutics Program, Children’s Hospital of Philadelphia (CHOP), said in a news release.

The approval in adolescents was based on the NETTER-P trial, which evaluated Lutathera in patients aged 12 to <18 years old with SSTR positive gastroenteropancreatic neuroendocrine tumors.

The study reported a safety profile consistent with the adult population studied in NETTER-1, the pivotal trial for approval of Lutathera in adults.

Additionally, Novartis in January 2024, reported data from the phase 3 NETTER-2 trial. In this trial, Lutathera plus long-acting release octreotide reduced the risk of disease progression or death by 72% as first-line therapy in patients with SSTR positive grade 2/3 advanced gastroenteropancreatic neuroendocrine tumors compared with high-dose octreotide alone. Data were presented at the 2024 American Society of Clinical Oncology (ASCO) Gastrointestinal (GI) Cancers Symposium.

In the NETTER-2 trial, most patients (88%) in the Lutathera arm received all four cycles of Lutathera treatment. The most common all-grade adverse events were nausea, diarrhea and abdominal pain, and the most common grade ≥3 adverse event was lymphocyte count decrease.