FDA approves lutetium Lu 177 dotatate for pediatric patients 12 years and older with GEP-NETS

Regulatory approval: On April 23, 2024, the FDA approved lutetium Lu 177 dotatate (Lutathera) for pediatric patients aged 12 years and older with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut NETs. The decision was supported by PK/dosimetry/safety data from the ongoing NETTER-P study and efficacy extrapolation from the adult NETTER-1 trial. A post-marketing requirement will assess long-term safety in adolescents.

FDA approval (April 23, 2024)

The FDA approved lutetium Lu 177 dotatate (Lutathera; Advanced Accelerator Applications USA, Inc., a Novartis company) for pediatric patients aged ≥12 years with somatostatin receptor (SSTR)-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs), including foregut, midgut, and hindgut NETs. Lutetium Lu 177 dotatate was previously approved for adults in 2018.

The agency stated this is the first FDA approval of a radiopharmaceutical for pediatric patients aged ≥12 years with SSTR-positive GEP-NETs.

Evidence base

Approval was supported by:

  • NETTER-P (NCT04711135): ongoing, international, multicenter, open-label, single-arm study in adolescents with locally advanced/inoperable or metastatic SSTR-positive GEP-NETs or pheochromocytoma/paraganglioma (PPGL); FDA cited pharmacokinetic, dosimetry, and safety data.
  • NETTER-1 (NCT01578239): randomized, multicenter, open-label, active-controlled trial (n=229) in adults with locally advanced/inoperable or metastatic SSTR-positive midgut carcinoid tumors; FDA cited extrapolation of efficacy outcomes supporting the pediatric indication.

Safety in NETTER-P was evaluated in 9 pediatric patients (including 4 with GEP-NETs); the adverse reaction profile was reported as similar to adults.

Dosing and follow-up

Recommended dose: 7.4 GBq (200 mCi) every 8 weeks (±1 week) for 4 doses. FDA issued a post-marketing requirement to assess long-term safety in adolescents.

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