marked.bio

RET inhibitor · small molecule

pralsetinib

5 active trials across 7 of our biomarkers, targeting RET. 1 active approval for a biomarker on marked.bio.

Also known as BLU-667, Gavreto.

Counts only trials that select patients by one of the biomarkers on marked.bio, not every trial of this drug.

Approvals and pivotal results

Biomarker-selected approvals in the United States and the European Union, with the pivotal trial's results and side effects where we have them. Results from different trials are not directly comparable.

TherapyBiomarkerTumourUnited StatesEuropean Union
pralsetinib
FDA, 2020
metastatic RET fusion-positive Accelerated approval 04/09/2020; regular approval 09/08/2023. Source
EU (EMA), 2021
advanced RET fusion-positive, not previously treated with a RET inhibitor EU conditional authorisation 18/11/2021; withdrawn 24/10/2024 at the holder's request (commercial reasons). Source
Gavreto 		RET fusion or mutation NSCLC Approved 2020 Withdrawn approved 2021

ARROW single-arm, 107 untreated; 130 after platinum
Response 78% (untreated); 63% (after platinum) (68-85; 54-71)Lasting 13.4 months (untreated); 38.8 months (after platinum)

Trial details and side effects
Patients
RET fusion-positive metastatic NSCLC (NCT03037385)
Most common side effects
musculoskeletal pain, constipation, hypertension, diarrhoea, fatigue, oedema, pyrexia, cough
Notes
FDA approval summary (2023); side effects are those in 25% or more of patients.
Source
FDA approval summary
pralsetinib
FDA, 2020
advanced or metastatic RET-mutant MTC Accelerated approval 01/12/2020; indication withdrawn (announced 30/06/2023) because the confirmatory trial was no longer feasible. Source
Gavreto 		RET fusion or mutation medullary thyroid Withdrawn approved 2020 Not approved

Biomarkers

BiomarkerActive trialsAll trialsFurthest active phase
RET fusion or mutation 5 8 Phase 4
BRAF V600E 3 3 Phase 4
NTRK1/2/3 fusion 3 3 Phase 4
ALK fusion 2 2 Phase 2
KRAS G12C 2 2 Phase 2
ROS1 fusion 2 2 Phase 2
EGFR exon 20 insertion 1 1 Phase 2

Sponsors

Industry sponsors leading active biomarker-selected trials of pralsetinib.

  1. Roche 1
  2. Rigel Pharmaceuticals 1

Trials

5 of 8 trials shown

  1. Pralsetinib DDI Study in Patients With Advanced or Metastatic Solid Tumors

    Recruiting Phase 4 Rigel Pharmaceuticals Started 2026Primary completion 2027-07 NCT07704658

    pralsetinib FDACYP3A4, CYP2C8, and CYP2C9 substrates, and hormonal contraceptive

    … has relapsed after, or is not responsive to, standard therapies and harbors an oncogenic RET fusion or mutation as determined by a validated test.

    RET fusion or mutationNSCLC (Non-small Cell Lung Cancer), Thyroid Cancer, Solid Tumor Malignancies, Advanced Malignancies

  2. Genotype-Driven Neoadjuvant Therapy for Locally Advanced Thyroid Cancer: A Real-World Cohort Study

    Not yet recruiting Phase 4 Fujian Medical University Started 2025Primary completion 2027-12 NCT07010393

    dabrafenib FDA EUtrametinib FDA EUselpercatinibpralsetiniblenvatiniblarotrectinibanlotinibpembrolizumabsintilimabcabozantinibBemosuzumab

    BRAF V600E mutation

    BRAF V600ERET fusion or mutationNTRK1/2/3 fusionThyroid Neoplasms

  3. Neoadjuvant Umbrella Trial for Patients With Unresectable Stage III NSCLC Harboring Rare Mutations.

    Recruiting Phase 2 Sun Yat-sen University Started 2024Primary completion 2027-11 NCT06563999

    sunvozertinib FDAcrizotinibpralsetiniblarotrectinibsavolitinibpyrotinibdabrafenibtrametinibglecirasibensartinib

    Subjects should have a rare mutation based on NGS, including mutations of EGFR exon20ins, ROS1 fusion, RET fusion, NTRK fusion, MET 14 exon, HER2, BRAF V600E, KRAS G12C, and ALK …

    EGFR exon 20 insertionKRAS G12CBRAF V600EALK fusionROS1 fusionRET fusion or mutationNTRK1/2/3 fusionLung Cancer Stage III, Mutation

  4. A Study of Multiple Therapies in Biomarker-selected Participants With Resectable Stages IB-III Non-small Cell Lung Cancer (NSCLC)

    Recruiting Phase 2 Roche Started 2020Primary completion 2026-11 NCT04302025

    alectinibentrectinibvemurafenibcobimetinibpralsetinibatezolizumabSBRTChemotherapydivarasib

    …on in ≥ 1% tumor cells as determined by Food and Drug Administration (FDA)-approved test, KRAS G12C mutation

    KRAS G12CBRAF V600EALK fusionROS1 fusionRET fusion or mutationNTRK1/2/3 fusionNon-small Cell Lung Cancer

  5. A Real-world Study of Pralsetinib Combined With Leucogen in the Treatment of RET Fusion-positive NSCLC

    Not yet recruiting No phase Fudan University Started 2026Primary completion 2027-12 NCT07418879

    pralsetinib FDALeucogen

    (3)RET fusion-positive;

    RET fusion or mutationNon-small Cell Lung Cancer, RET Fusion, Pralsetinib