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Therapy Name Pralsetinib
Synonyms
Therapy Description

Gavreto (pralsetinib) is a small molecule inhibitor that selectively targets activated Ret, which may result in antitumor activity including inhibition of tumor growth and tumor regression (PMID: 29657135). Gavreto (pralsetinib) is FDA approved for use in patients with metastatic RET fusion-positive non-small cell lung cancer, in adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer, and in adult and pediatric patients 12 years of age and older with RET fusion-positive thyroid cancer who are radioactive iodine-refractory (FDA.gov).

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Pralsetinib Gavreto BLU667|BLU-667 RET Inhibitor 52 Gavreto (pralsetinib) is a small molecule inhibitor that selectively targets activated Ret, which may result in antitumor activity including inhibition of tumor growth and tumor regression (PMID: 29657135). Gavreto (pralsetinib) is FDA approved for use in patients with metastatic RET fusion-positive non-small cell lung cancer, in adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer, and in adult and pediatric patients 12 years of age and older with RET fusion-positive thyroid cancer who are radioactive iodine-refractory (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
RET fusion differentiated thyroid gland carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for adult or pediatric patients 12 years or older with radioactive iodine-refractory, advanced or metastatic differentiated thyroid gland carcinoma harboring RET fusions (PMID: 31549998, PMID: 35491008; ESMO.org). 35491008 31549998 detail...
RET fusion thyroid gland follicular carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for patients with follicular thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET over exp pulmonary neuroendocrine tumor predicted - sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited proliferation of a lung neuroendocrine tumor cell line with high RET expression in 3D spheroid culture (Cancer Res (2023) 83 (7_Supplement): 4039). detail...
RET D631Y Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET D631Y in culture (PMID: 36166639). 36166639
RET D567N Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and viability in transformed cells expressing RET D567N in culture and inhibited tumor growth in a cell line xenograft model (PMID: 36166639). 36166639
RET C634R Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and viability in transformed cells expressing RET C634R in culture and inhibited tumor growth in a cell line xenograft model (PMID: 36166639). 36166639
RET fusion thyroid gland anaplastic carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as systemic therapy for patients with anaplastic thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET fusion thyroid cancer sensitive Pralsetinib FDA approved Actionable In a Phase I/II trial (ARROW) that supported FDA approval, Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 89% (8/9) in patients with advanced or metastatic RET fusion-positive thyroid cancer, with a median duration of response of 14.5 months, and 67% of responding patients continuing treatment (PMID: 34118198; NCT03037385). 34118198 detail...
RET fusion thyroid gland Hurthle cell carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for patients with thyroid Hurthle cell carcinoma harboring RET fusions (NCCN.org). detail...
HRAS A59T RET A883F RET A883T thyroid gland medullary carcinoma predicted - resistant Pralsetinib Case Reports/Case Series Actionable In a retrospective analysis, a medullary thyroid carcinoma patient harboring RET A883T progressed on treatment with Gavreto (pralsetinib) and was found to have acquired RET A883F and HRAS A59T (PMID: 38127829; NCT03157128, NCT03037385, NCT03780517). 38127829
RET M918T thyroid gland medullary carcinoma sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I trial, a patient with sporadic medullary thryoid cancer harboring RET M918T demonstrated a partial response, with maximal tumor reduction of 47% following treatment with Gavreto (pralsetinib) (PMID: 29657135; NCT03037385). 29657135
RET M918T thyroid gland medullary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 65% (51/79, 5% complete response, 59% partial response) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations, 61% of the patients harbored RET M918T (Ann Oncol. Vol 31, Supplement 4, S1084, Sep 1, 2020; NCT03037385). detail...
RET rearrange lung non-small cell carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as a first-line and subsequent therapy for patients with advanced or metastatic non-small cell lung cancer harboring RET rearrangements (NCCN.org). detail...
RET Y806C RET M918T thyroid gland medullary carcinoma predicted - resistant Pralsetinib Case Reports/Case Series Actionable In a retrospective analysis, a medullary thyroid carcinoma patient harboring RET M918T progressed on treatment with Gavreto (pralsetinib) and was found to have acquired RET Y806C (PMID: 38127829; NCT03157128, NCT03037385, NCT03780517). 38127829
RET fusion intrahepatic cholangiocarcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as primary (category 2B) and subsequent-line therapy (category 2B) for patients with unresectable or metastatic biliary tract cancer harboring RET fusions, including intrahepatic cholangiocarcinoma (NCCN.org). detail...
RET fusion intrahepatic cholangiocarcinoma sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in a partial response in a patient with RET fusion-positive intrahepatic cholangiocarcinoma, with a duration of response of 7.5 months (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET C630del Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) treatment inhibited Ret phosphorylation and downstream signaling and resulted in decreased cell viability and colony formation in cells expressing RET C630del in culture (PMID: 35689816). 35689816
EML4 - RET lung adenocarcinoma sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, a patient-derived lung adenocarcinoma cell line harboring EML4-RET was sensitive to treatment with Gavreto (pralsetinib), demonstrating decreased cell proliferation in culture, and inhibition of tumor growth in a xenograft model (PMID: 33795352). 33795352
RET fusion gallbladder cancer sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as primary (category 2B) and subsequent-line therapy (category 2B) for patients with unresectable or metastatic biliary tract cancer harboring RET fusions, including gallbladder cancer (NCCN.org). detail...
RET fusion extrahepatic bile duct carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as primary (category 2B) and subsequent-line therapy (category 2B) for patients with unresectable or metastatic biliary tract cancer harboring RET fusions, including extrahepatic cholangiocarcinoma (NCCN.org). detail...
RET D898_E901del Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Erk phosphorylation and growth of transformed cells expressing RET D898_E901del in culture (PMID: 32284345). 32284345
RET C634X thyroid gland medullary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 65% (51/79, 5% complete response, 59% partial response) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations, 28% of the patients harbored RET C634X (Ann Oncol. Vol 31, Supplement 4, S1084, Sep 1, 2020; NCT03037385). detail...
RET D584N Advanced Solid Tumor sensitive Pralsetinib Preclinical - Biochemical Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling in transformed cells expressing RET D584N in culture (PMID: 36166639). 36166639
RET C634W thyroid gland medullary carcinoma sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and proliferation of a medullary thyroid cancer cell line harboring RET C634W in culture, and inhibited tumor growth in xenograft models (PMID: 29657135). detail... 29657135
RET mutant thyroid gland medullary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines (category 2B) for patients with medullary thyroid carcinoma harboring RET mutations (NCCN.org). detail...
RET mutant thyroid gland medullary carcinoma sensitive Pralsetinib FDA approved Actionable In a Phase I/II trial (ARROW) that supported FDA approval, Gavreto (pralsetinib) treatment was well-tolerated and resulted in an overall response rate (ORR) of 60% (3/55) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations who received prior treatments, ORR was 60% (32/53) in patients with prior therapies and 71% (15/21) in treatment-naive patients (PMID: 34118198; NCT03037385). 34118198 detail...
RET mutant thyroid gland medullary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines for adult or pediatric patients 12 years or older with advanced or metastatic medullary thyroid gland carcinoma harboring RET mutations (PMID: 31549998, PMID: 35491008; ESMO.org). 31549998 detail... 35491008
RET C620R Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET C620R in culture (PMID: 36166639). 36166639
RET D571N Advanced Solid Tumor sensitive Pralsetinib Preclinical - Biochemical Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling in transformed cells expressing RET D571N in culture (PMID: 36166639). 36166639
RET fusion pancreatic cancer predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in a partial response in 2 of 2 patients with RET fusion positive pancreatic cancer, with a duration of response of 5.5 and 7.4 months, respectively (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET T930K Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited growth of transformed cells expressing RET T930K in culture (PMID: 32284345). 32284345
RET fusion colon cancer predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in stable disease in 2 patients with RET fusion-positive colon cancer, with a duration of response of 7.3 and 9.3 months, respectively (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET fusion thyroid gland medullary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as systemic therapy for patients with medullary thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET V591_G607del RET L1016S thyroid gland medullary carcinoma predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a clinical case study, Gavreto (pralsetinib) treatment resulted in a partial response lasting more than 1 year in a patient with medullary thyroid carcinoma harboring RET V591_G607del and germline RET L1016S (PMID: 36053791). 36053791
RET fusion lung non-small cell carcinoma sensitive Pralsetinib Clinical Study Actionable In a retrospective study, Gavreto (pralsetinib) treatment resulted in an objective response rate (ORR) of 61% (39/59, 2 complete responses (CR), 37 partial responses (PR)) and a disease control rate (DCR) of 79.7% in non-small cell lung cancer patients harboring RET fusions, and an intracranial ORR of 66.7% (4/6, 3 CR and 1 PR), an intracranial DCR of 100% in patients with measurable brain metastases (PMID: 36379124). 36379124
RET fusion lung non-small cell carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as a first-line therapy for patients with metastatic non-small cell lung cancer harboring RET fusions (PMID: 36872130; ESMO.org). detail... 36872130
RET fusion lung non-small cell carcinoma sensitive Pralsetinib FDA approved - On Companion Diagnostic Actionable In a Phase I/II trial (ARROW) that supported FDA approval, Gavreto (pralsetinib) treatment was well-tolerated, resulting in an objective response rate of 61% (53/87) and 70% (19/27) in previously treated and treatment-naive patients with RET fusion-positive non-small cell lung cancer, respectively (PMID: 34118197; NCT03037385). detail... 34118197 detail...
RET E574D Advanced Solid Tumor sensitive Pralsetinib Preclinical - Biochemical Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling in transformed cells expressing RET E574D in culture (PMID: 36166639). 36166639
RET E632_L633del Advanced Solid Tumor conflicting Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, a cell line expressing RET E632_L633del was less sensitive to Gavreto (pralsetinib) than cells expressing wild-type RET in culture (PMID: 36416226). 36416226
RET E632_L633del Advanced Solid Tumor conflicting Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET E632_L633del in culture (PMID: 36166639). 36166639
RET D567Y Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell line xenograft Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Ret signaling and viability in transformed cells expressing RET D567Y in culture and inhibited tumor growth in a cell line xenograft model (PMID: 36166639). 36166639
RET L629P RET D631_R635delinsG RET V637R thyroid gland medullary carcinoma predicted - sensitive Pralsetinib Case Reports/Case Series Actionable In a Phase I trial, a patient with medullary thryoid cancer harboring RET D631_R635delinsG, L629P, and V637R demonstrated a partial response, with maximal tumor reduction of 47% at 10 months, following treatment with Gavreto (pralsetinib) (PMID: 29657135; NCT03037385). 29657135
RET G607C Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET G607C in culture (PMID: 36166639). 36166639
RET V804X thyroid gland medullary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, and resulted in an overall response rate (ORR) of 65% (51/79, 5% complete response, 59% partial response) in patients with advanced or metastatic medullary thyroid cancer harboring RET mutations, 4% of the patients harbored RET V804X (Ann Oncol. Vol 31, Supplement 4, S1084, Sep 1, 2020; NCT03037385). detail...
RET fusion thyroid gland papillary carcinoma sensitive Pralsetinib Guideline Actionable Gavreto (pralsetinib) is included in guidelines as systemic therapy for patients with papillary thyroid carcinoma harboring RET fusions (NCCN.org). detail...
RET fusion thyroid gland papillary carcinoma sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well-tolerated, resulted in an objective response rate of 75% (9/12) and a median duration of response of 14.5 months in patients with RET fusion positive papillary thyroid cancer (J Clin Oncol 38, no. 15_suppl (May 20, 2020) 109-109; NCT03037385). detail...
RET D631_L633delinsE Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET D631_L633delinsE in culture (PMID: 36166639). 36166639
RET fusion Advanced Solid Tumor predicted - sensitive Pralsetinib Phase Ib/II Actionable In a Phase I/II trial (ARROW), Gavreto (pralsetinib) treatment was well tolerated in patients with advanced solid tumors harboring RET fusions, and led to an overall response rate of 57% (13/23, 3 complete and 10 partial responses), a clinical benefit rate of 70% (16/23), a disease control rate of 83% (19/23), a median duration of response of 11.7 months, a median progression-free survival of 7.4 months, and a median overall survival of 13.6 months (PMID: 35962206; NCT03037385). 35962206
RET M918T Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited Erk phosphorylation and growth of transformed cells expressing RET M918T in culture (PMID: 32284345). 32284345
RET M918T Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited viability in transformed cells expressing RET M918T in culture (PMID: 36166639). 36166639
RET T930P Advanced Solid Tumor sensitive Pralsetinib Preclinical - Cell culture Actionable In a preclinical study, Gavreto (pralsetinib) inhibited growth of transformed cells expressing RET T930P in culture (PMID: 32284345). 32284345

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT04222972 Phase III Carboplatin + Pembrolizumab + Pemetrexed Disodium Carboplatin + Paclitaxel + Pembrolizumab Carboplatin + Nab-paclitaxel + Pembrolizumab Pralsetinib Cisplatin + Gemcitabine Carboplatin + Gemcitabine Cisplatin + Pemetrexed Disodium Cisplatin + Pembrolizumab + Pemetrexed Disodium Carboplatin + Pemetrexed Disodium A Study of Pralsetinib Versus Standard of Care for First-Line Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC) (AcceleRET-Lung) Active, not recruiting USA | NLD | ITA | GBR | FRA | ESP | DEU | BEL | AUS 14
NCT04302025 Phase II Cobimetinib + Vemurafenib Entrectinib Atezolizumab Alectinib Pralsetinib A Study of Multiple Therapies in Biomarker-Selected Patients With Resectable Stages IB-III Non-Small Cell Lung Cancer Recruiting USA 0
NCT05170204 Phase III Durvalumab Alectinib Pralsetinib Entrectinib A Study Evaluating the Efficacy and Safety of Multiple Therapies in Cohorts of Participants With Locally Advanced, Unresectable, Stage III Non-Small Cell Lung Cancer (NSCLC) Recruiting USA | NZL | ITA | GBR | FRA | ESP | DEU | CAN | BEL | AUS 17
NCT03037385 Phase Ib/II Pralsetinib Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Participants With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW) Completed USA | NLD | ITA | GBR | FRA | ESP | DEU | BEL 5
NCT04760288 Phase III Cabozantinib Pralsetinib Vandetanib A Study of Pralsetinib Versus Standard of Care (SOC) for Treatment of RET-Mutated Medullary Thyroid Cancer (MTC). (AcceleRET-MTC) Withdrawn ESP 0


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