|Drug class||Tyrosine kinase inhibitor|
|Main uses||Non-small cell lung cancer (NSCLC), thyroid cancer|
|Side effects||Constipation, high blood pressure, tiredness, muscle pain, low blood cells, liver problems, low calcium|
|Typical dose||400 mg OD|
|Chemical and physical data|
|Molar mass||533.612 g·mol−1|
|3D model (JSmol)|
Pralsetinib, sold under the brand name Gavreto, is a medication used to treat non-small cell lung cancer (NSCLC) and thyroid cancer. It is used in advanced cases which are RET fusion-positive. It is taken by mouth.
Common side effects include constipation, high blood pressure, tiredness, muscle pain, low blood cells, liver problems, and low calcium. Other side effects may include pneumonitis, bleeding, tumor lysis syndrome, and poor wound healing. Use in pregnancy may harm the baby. It is a tyrosine kinase inhibitor.
It is generally taken at a dose of 400 mg once daily.
Efficacy was investigated in a multicenter, open-label, multi-cohort clinical trial (ARROW, NCT03037385) with 220 participants aged 26-87 whose tumors had RET alterations. Identification of RET gene alterations was prospectively determined in local laboratories using either next generation sequencing, fluorescence in situ hybridization, or other tests. The main efficacy outcome measures were overall response rate (ORR) and response duration determined by a blinded independent review committee using RECIST 1.1. The trial was conducted at sites in the United States, Europe and Asia.
Efficacy for RET fusion-positive NSCLC was evaluated in 87 participants previously treated with platinum chemotherapy. The ORR was 57% (95% CI: 46%, 68%); 80% of responding participants had responses lasting 6 months or longer. Efficacy was also evaluated in 27 participants who never received systemic treatment. The ORR for these participants was 70% (95% CI: 50%, 86%); 58% of responding participants had responses lasting 6 months or longer.
The US Food and Drug Administration (FDA) granted the application for pralsetinib priority review, orphan drug, and breakthrough therapy designationsand granted approval of Gavreto to Blueprint Medicines.
Society and culture
On 16 September 2021, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Gavreto, intended for the treatment of people with rearranged during transfection (RET)-fusion positive non-small cell lung cancer (NSCLC). The applicant for this medicinal product is Roche Registration GmbH.
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- Clinical trial number NCT03037385 for "Phase 1/2 Study of the Highly-selective RET Inhibitor, Pralsetinib (BLU-667), in Patients With Thyroid Cancer, Non-Small Cell Lung Cancer, and Other Advanced Solid Tumors (ARROW)" at ClinicalTrials.gov
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