FDA Approval Summary: Selpercatinib for the Treatment of Lung and Thyroid Cancers with RET Gene Mutations or Fusions
Top Cited Papers
- 15 April 2021
- journal article
- research article
- Published by American Association for Cancer Research (AACR) in Clinical Cancer Research
- Vol. 27 (8), 2130-2135
- https://doi.org/10.1158/1078-0432.CCR-20-3558
Abstract
On May 8, 2020, the FDA granted accelerated approval to selpercatinib for (i) adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC), (ii) adult and pediatric patients >= 12 years of age with advanced or metastatic RET-mutant medullary thyroid cancer who require systemic therapy, and (iii) adult and pediatric patients >= 12 years of age with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine refractory (if radioactive iodine is appropriate). Approval was granted on the basis of the clinically important effects on the overall response rate (ORR) with prolonged duration of responses observed in a multicenter, open-label, multicohort clinical trial (LIBRETTO-001, NCT03157128) in patients whose tumors had RET alterations. ORRs within the approved patient populations ranged from 64% [95% confidence interval (CI), 54-73] in prior platinum-treated RET fusion-positive NSCLC to 100% (95% CI, 63-100) in systemic therapy-naive RET fusion-positive thyroid cancer, with the majority of responders across indications demonstrating responses of at least 6 months. The product label includes warnings and precautions for hepatotoxicity, hypertension, QT interval prolongation, hemorrhagic events, hypersensitivity, risk of impaired wound healing, and embryo-fetal toxicity. This is the first approval of a drug specifically for patients with RET alterations globally.Other Versions
This publication has 20 references indexed in Scilit:
- Lung cancer — major changes in the American Joint Committee on Cancer eighth edition cancer staging manualCA: A Cancer Journal for Clinicians, 2017
- Correlative analyses of RET and RAS mutations in a phase 3 trial of cabozantinib in patients with progressive, metastatic medullary thyroid cancerCancer, 2016
- Benefit-Risk Summary of Crizotinib for the Treatment of Patients With ROS1 Alteration-Positive, Metastatic Non-Small Cell Lung CancerThe Oncologist, 2016
- A comprehensive overview of the role of the RET proto-oncogene in thyroid carcinomaNature Reviews Endocrinology, 2016
- Epidemiology and Clinical Presentation of Medullary Thyroid CarcinomaPublished by Springer Science and Business Media LLC ,2015
- American Thyroid Association Guidelines for Management of Patients with Anaplastic Thyroid CancerThyroid®, 2012
- Vandetanib in Patients With Locally Advanced or Metastatic Medullary Thyroid Cancer: A Randomized, Double-Blind Phase III TrialJournal of Clinical Oncology, 2012
- An evidence‐based review of poorly differentiated thyroid cancerWorld Journal of Surgery, 2007
- Anaplastic thyroid carcinoma: three protocols combining doxorubicin, hyperfractionated radiotherapy and surgeryBritish Journal of Cancer, 2002
- Long-term impact of initial surgical and medical therapy on papillary and follicular thyroid cancerAmerican Journal Of Medicine, 1994