Ga-68 DOTATATE gets orphan drug status for neuroendocrine cancer imaging

Neuroendocrine tumor imaging just got a boost with both U.S Food and Drug Administration (FDA) and European Medicines Agency (EMA) orphan drug designation for gallium-68 DOTATATE, used in PET imaging of neuroendocrine cancer.

The orphan drug status was announced earlier this month by Advanced Accelerator Applications (AAA) based in Saint Genis Pouilly, France. Among a group of gallium-68 PET agents for neuroendocrine cancer imaging, DOTATATE is short for the very unwieldy compound title of gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide. DOTATATE is an investigational diagnostic somatostatin analogue peptide PET agent among a group of peptide agents used to track down neuroendocrine tumors among the myriad organs that interact with the neuroendocrine system, especially those of the digestive tract.

“GEP-NETs constitute a life-threatening disease and effective patient management requires accurate diagnostic tools,” said AAA CEO tefano Buono. “The orphan drug designation of AAA's Gallium-68 DOTATATE will accelerate the development of this agent and hopefully allow it to be available to patients in the next few years.”

Orphan drug designation is intended to support the development of drugs for relatively rare life-threatening illnesses. FDA drug orphan status in particular requires that a drug treat a disease that affects fewer than 200,000 Americans. DOTATATE’s new drug status could potentially fast-track approval for PET imaging of neuroendocrine cancer and open doors to more resources toward that end.