First radioactive drug approved for patients 12 years and up with SSTR-positive GEP-NETs

First radioactive drug approved for patients 12 years and up with SSTR-positive GEP-NETs | Image Credit: © Calin - © Calin - stock.adobe.com.

The FDA has approved lutetium Lu 177 dotatate (Lutathera; Advanced Accelerator Applications USA [Novartis]) for patients with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) aged 12 years and older, according to a press release from the federal agency.¹

The indication includes foregut, midgut, and hindgut neuroendocrine tumors. The decision by the FDA makes lutetium Lu 177 dotatate the first FDA approval of a radioactive drug, or radiopharmaceutical for the aforementioned population.¹

The approval was based on pharmacokinietic, dosimetry, and safety data demonstrated in the ongoing, multicenter, international, open-label, single-arm phase 2 NETTER-P trial (NCT04711135). The study enrolled 11 patients aged 12 to <18 years.^1,2

The study schedule consists of an up to 2-week screening period, followed by 4 treatment administrations at 8-week intervals, and a 5-year follow-up period.²

The approval was also based on efficacy outcomes observed in the NETTER-1 study (NCT01578239) that featured 229 patients aged 18 years and older in a randomized, multicenter, open-label, active-controlled, phase 3 design.^1,3

In the NETTER-P study, safety was evaluated in 9 pediatric patients,4 of which with GEP-NETs. Major outcome measures were "absorbed radiation doses in target organs and incidence of adverse reactions after the first treatment cycle," the FDA stated.¹

The recommended dose for lutetium Lu 177 dotatate is GBq (200 mCi) every 8 weeks (± 1 week) for a total of 4 doses. Premedications and concomitant medications should be administered as recommended.¹

The FDA noted that a post-marketing requirement was issued to assess long-term safety of the radioactive drug in adolescents.¹

The NETTER-P study was conducted as part of a pediatric Written Request under the Pharmaceuticals for Children Act. The application for lutetium Lu 177 dotatate was granted Priority Review and Orphan Drug Designation by the FDA.¹

References:

1. FDA approves lutetium Lu 177 dotatate for pediatric patients 12 years and older with GEP-NETS. FDA. Press release. April 23, 2024. Accessed April 23, 2024. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-lutetium-lu-177-dotatate-pediatric-patients-12-years-and-older-gep-nets

2. Study to evaluate safety and dosimetry of Lutathera in adolescent patients with GEP-NETs and PPGLs. ClinicalTrials.gov. Updated February 15, 2024. Accessed April 23, 2024. https://clinicaltrials.gov/study/NCT04711135

3. A Study Comparing Treatment With 177Lu-DOTA0-Tyr3-Octreotate to Octreotide LAR in Patients With Inoperable, Progressive, Somatostatin Receptor Positive Midgut Carcinoid Tumours (NETTER-1). ClinicalTrials.gov. Updated April 4, 2022. Accessed April 23, 2024. https://classic.clinicaltrials.gov/ct2/show/NCT01578239