Novartis has received U.S. FDA approval for lutetium (177Lu) oxodotreotide (Lutathera) for the treatment of somatostatin receptor positive GEP-NETs.
This morning, Novartis AG announced that Advanced Accelerator Applications, a subsidiary of Novartis Groupe S.A., has received U.S. Food and Drug Administration (FDA) approval for lutetium (177Lu) oxodotreotide (Lutathera) for the treatment of somatostatin receptor positive gastroenteropancreatic neuroendocrine tumors (NETs) (GEP-NETs).
The list of NETs for which the drug is now approved includes: foregut, midgut, and hindgut neuroendocrine tumors in adults. The approval marks the first of its kind of a Peptide Receptor Radionuclide Therapy (PRRT), and expands Novartis’ NET portfolio. In August, the FDA accepted the resubmission of the New Drug Application for Lutathera
NETs are a rare cancer type, originating in the body’s neuroendocrine cells; often in the gastrointestinal tract, lungs or pancreas. Symptoms can vary among patients, however, most develop symptoms arising from an excessive production of hormones. Lutathera is a form of targeted therapy in which the radiolabeled drug binds to somatostatin receptors present in GEN-NETs.
“The approval of Lutathera marks an important achievement and an innovation greatly needed for the NET cancer community,” said Susanne Schaffert, Ph.D., Chairperson and President, Advanced Accelerator Applications in a press release. “For 30 years, Novartis has supported the NET community with the development of therapeutics in NET and carcinoid syndrome. I cannot think of a better way to commemorate the joining of two organizations and our future together as we advance new nuclear medicine therapeutics in NET as well as across other tumor types.”
Today’s approval is based on results from a randomized pivotal Phase 3 study (NETTER-1) that compared treatment with the drug plus best standard-of-care (octreotide LAR 30mg every 4 weeks) to 60mg of octreotide LAR at the same dosage in patients with inoperable midgut NETs progressing under standard dose octreotide LAR treatment and overexpressing somatostatin receptors. Additional safety and efficacy data from a single-institution, single-arm, open-label trial conducted by Erasmus Medical Center in Rotterdam, Netherlands in more than 1,200 patients was also taken into consideration.
"This approval provides another treatment choice for patients with these rare cancers. It also demonstrates how the FDA may consider data from therapies that are used in an expanded access program to support approval for a new treatment," said Richard Pazdur, M.D., director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research in a second press release.
The NETTER-1 study met its primary endpoint, exhibiting a 79% reduction in risk of disease progression or death, however, median progression-free survival (PFS) was not reached in the Lutathera arm. The most common Grade 3-4 adverse reactions occurring in patients included: lymphopenia, increased gamma-glutamyl transferase, vomiting, nausea, and elevated aspartate aminotransferase, and increased alanine aminotransferase, hyperglycemia, and hypokalemia.
Lutathera comes with a warning stating that treatment with it can contribute to a patient’s overall long-term radiation exposure and is associated with an increased risk for cancer.
At the 2015 North American Neuroendocrine Tumor Society (NANETS) Annual Symposium, Rare Disease Report spoke with Jonathan R. Strosberg, M.D., medical oncologist, of the Department of Gastrointestinal Oncology at the Moffitt Cancer Center about the Phase 3 NETTER-1 trial.