Pralsetinib
Pralsetinib, sold under the brand name Gavreto, is a medication approved for RET mutation-positive medullary thyroid cancer and RET fusion-positive differentiated thyroid cancer refractory to radioactive iodine therapy. Pralsetinib is a tyrosine kinase inhibitor. It is taken by mouth.
The most common adverse reactions include increased aspartate aminotransferase, decreased hemoglobin, decreased lymphocytes, decreased neutrophils, increased alanine aminotransferase, increased creatinine, increased alkaline phosphatase, fatigue, constipation, musculoskeletal pain, decreased calcium, hypertension, decreased sodium, decreased phosphate, and decreased platelets.
Pralsetinib was approved for medical use in the United States in September 2020, and in the European Union in November 2021.
Medical uses
Pralsetinib is indicated for the treatment of adults with metastatic RET fusion-positive non-small cell lung cancer as detected by an FDA approved test.History
Efficacy was investigated in a multicenter, open-label, multi-cohort clinical trial 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 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% ; 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% ; 58% of responding participants had responses lasting 6 months or longer.
The US Food and Drug Administration granted the application for pralsetinib priority review, orphan drug, and breakthrough therapy designationsand granted approval of Gavreto to Blueprint Medicines.