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Molecular Profile RET mutant
Therapy Selpercatinib
Indication/Tumor Type medullary thyroid carcinoma
Response Type sensitive

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
RET mutant medullary thyroid carcinoma sensitive Selpercatinib FDA approved - Has Companion Diagnostic Actionable In a Phase I/II trial (LIBRETTO-001) that supported FDA approval, Retevmo (selpercatinib) treatment resulted in an objective response rate (ORR) of 69% (38/55), with five complete and 33 partial responses, in adult and pediatric patients of 12 years and older with medullary thyroid cancer harboring RET mutations who were previously treated, while patients who had not been previously treated demonstrated an ORR of 73% (64/88), with ten complete and 54 partial responses (PMID: 32846061; NCT03157128). detail... detail... 32846061
RET mutant medullary thyroid carcinoma sensitive Selpercatinib Guideline Actionable Retevmo (selpercatinib) is included in guidelines for adult or pediatric patients 12 years or older with advanced medullary thyroid gland carcinoma harboring RET mutations (PMID: 31549998, PMID: 35491008; ESMO.org). 31549998 detail... 35491008
RET mutant medullary thyroid carcinoma sensitive Selpercatinib Guideline Actionable Retevmo (selpercatinib) is included in guidelines for patients with medullary thyroid carcinoma harboring RET mutations (NCCN.org). detail...
RET mutant medullary thyroid carcinoma sensitive Selpercatinib FDA approved - Has 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...
PubMed Id Reference Title Details
NCCN.org Full reference...
ESMO Clinical Practice Guidelines Full reference...
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...
(35491008) ESMO Clinical Practice Guideline update on the use of systemic therapy in advanced thyroid cancer. Full reference...
(31549998) Thyroid cancer: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up†. 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...