Advanced Accelerator Applications receives FDA approval for Lutathera for treatment of gastroenteropancreatic neuroendocrine tumors.
Advanced Accelerator Applications, a subsidiary of Novartis Groupe S.A., has received U.S. Food and Drug Administration (FDA) approval of its new drug application (NDA) for Lutathera (lutetium Lu 177 dotatate*) for the treatment of somatostatin receptor positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut neuroendocrine tumors, in adults.
Lutathera, which received orphan drug designation from the FDA, is a first-in-class drug and the first available FDA-approved Peptide Receptor Radionuclide Therapy (PRRT), a form of treatment comprising of a targeting molecule that carries a radioactive component.
The approval was based on a Phase 3 study which demonstrated a 79 percent reduction in the risk of disease progression or death within the Lutathera plus best standard of care arm (octreotide LAR 30mg every four weeks) compared to 60 mg of octreotide LAR alone (hazard ratio 0.21, 95% CI; 0.13-0.32; p<00.001).
Novartis recently completed a successful tender offer for Advanced Accelerator Applications to become a subsidiary within the Novartis Group.
“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.
NETs are rare tumors originating in the neuroendocrine cells of numerous organs, including the gastrointestinal tract, pancreas, and lung. Some patients develop symptoms arising from the excessive production of hormones by neuroendocrine tumor cells, while others remain clinically silent for years.
The estimated incidence, or rate of new cases, of NETs in the United States is approximately 6.98/100,000 per year, while the estimated prevalence for 2014, based on the National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) database, was 171,321.1 Patient survival with advanced GEP-NETs depends on stage and histology. Patients with well- and moderately-differentiated tumors and distant metastases have a 5-year survival probability of 35 percent.2
The approval of Lutathera is based on results of a randomized pivotal Phase 3 study, NETTER-1 that compared treatment using Lutathera plus best standard of care (octreotide LAR 30mg every four weeks) to 60 mg of octreotide LAR, also dosed every four weeks, in patients with inoperable midgut NETs progressing under standard dose octreotide LAR treatment and overexpressing somatostatin receptors, as well as a subset of efficacy and safety data from an international, single-institution, single-arm, open-label trial conducted by Erasmus Medical Center in Rotterdam, Netherlands in more than 1,200 patients with somatostatin receptor positive tumors.
* USAN: lutetium Lu 177 dotatate/INN: lutetium (177Lu) oxodotreotide
1 Dasari A, Shen C, Halperin D, Zhao B, Zhou S, Xu Y, Shih T, Yao JC. Trends in the incidence, prevalence, and survival outcomes in patients with neuroendocrine tumors in the United States. JAMA Oncol. 2017; 3(10):1335-1342.
2 Yao JC, Hassan M, Phan A, et al. One hundred years after “carcinoid”: epidemiology of and prognostic factors for neuroendocrine tumors in 35,825 cases in the United States. J Clin Oncol. 2008;26:3063-3072.
(Source: Novartis Pharmaceuticals Corp.)
Filed Under: Drug Discovery