Profile Response Detail

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Molecular Profile	Indication/Tumor Type	Response Type	Therapy Name	Approval Status	Evidence Type	Efficacy Evidence	References
RET fusion	thyroid cancer	sensitive	Selpercatinib	FDA approved - On Companion Diagnostic	Actionable	In a Phase I/II trial (LIBRETTO-121) that supported FDA approval, Retevmo (selpercatinib) treatment was safe in pediatric patients of 2 years or older with solid tumors (8 medullary thyroid cancer, 2 papillary thyroid cancer, 1 osteosarcoma) harboring RET fusion (n=2) or mutations (n=9), and resulted in unconfirmed partial responses in 4 patients, stable disease in 6 patients (two lasting >/=16 weeks) (JCO 39, 10009-10009(2021); NCT03899792).	detail... detail... detail...
RET fusion	thyroid cancer	sensitive	Selpercatinib	FDA approved - On Companion Diagnostic	Actionable	In a Phase I/II trial (LIBRETTO-001) that supported FDA approval, Retevmo (selpercatinib) treatment resulted in an objective response rate of 79% (15/19), including one complete and 14 partial responses, with a median response duration of 18.4 months, and median progression-free survival of 20.1 months in adult and pediatric patients 12 years and older with RET fusion-positive thyroid cancer who were previously treated (PMID: 32846061; NCT03157128).	detail... detail... 32846061

PubMed Id	Reference Title	Details
		Full reference...
		Full reference...
	Retevmo (selpercatinib) FDA Drug Label	Full reference...
	Retevmo (selpercatinib) FDA Drug Label	Full reference...
(32846061)	Efficacy of Selpercatinib in RET-Altered Thyroid Cancers.	Full reference...
	Oral selpercatinib in pediatric patients (pts) with advanced RET-altered solid or primary CNS tumors: Preliminary results from the phase 1/2 LIBRETTO-121 trial.	Full reference...