The Food and Drug Administration has recently approved lutetium Lu 177 dotatate (Lutathera, Advanced Accelerator Applications USA, Inc., a Novartis company) for pediatric patients aged 12 and above diagnosed with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), which include foregut, midgut, and hindgut neuroendocrine tumors. This approval, granted in 2018 for adults, marks the first FDA approval of a radioactive drug, or radiopharmaceutical, for this specific pediatric patient population.
Image Source: Vinoth Khandelwal
The approval of Lutathera dotatate for pediatric patients was based on data from NETTER-P (NCT04711135), an ongoing international study evaluating its pharmacokinetics (PK), dosimetry, and safety in adolescents with locally advanced/inoperable or metastatic SSTR-positive GEP-NETs or pheochromocytoma/paraganglioma (PPGL). Additionally, efficacy outcomes observed in NETTER-1 (NCT01578239), a trial involving adults with midgut carcinoid tumors, supported this pediatric indication.
Safety evaluation in pediatric patients, including those with GEP-NETs, was conducted in NETTER-P, with a profile similar to that observed in adults. The recommended dose of Lutathera for pediatric patients is 7.4 GBq (200 mCi) every 8 weeks for a total of 4 doses, with premedications and concomitant medications administered as recommended. A post-marketing requirement has been issued to assess the long-term safety of the drug in adolescents.
NETTER-P was conducted under a pediatric Written Request (WR) as part of the Best Pharmaceuticals for Children Act (BPCA), granting priority review and orphan drug designation. FDA expedited programs, outlined in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics, facilitated this process.
Dr. Vinoth Khandelwal, Ph.D
