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Selpercatinib: First Approval

Anthony Markham1

  • 1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. dru@adis.com.

Drugs
|June 20, 2020
PubMed

Insights

Selpercatinib, a RET inhibitor, has gained FDA approval for treating specific lung and thyroid cancers with RET alterations. This marks a significant milestone in targeted cancer therapy development.

Area of Science:

  • Oncology
  • Pharmacology
  • Molecular Biology

Background:

  • Selpercatinib is a targeted therapy developed by Loxo Oncology.
  • It functions as a potent inhibitor of the rearranged during transfection (RET) receptor tyrosine kinase.
  • This drug is designed to treat various cancers that exhibit specific RET alterations.

Purpose of the Study:

  • To summarize the key developmental milestones of selpercatinib.
  • To highlight the clinical trial data leading to its approval.
  • To document the journey of selpercatinib from development to its first regulatory approval.

Main Methods:

  • Review of preclinical and clinical data from the LIBRETTO-001 trial.
  • Analysis of regulatory submission and approval processes.
  • Summary of developmental pathway and therapeutic application.

Main Results:

  • Selpercatinib demonstrated efficacy in patients with RET alterations.
  • The US FDA approved selpercatinib for specific indications.
  • Approved indications include RET fusion-positive non-small-cell lung cancer, RET fusion-positive thyroid cancer, and RET-mutant medullary thyroid cancer.

Conclusions:

  • Selpercatinib's approval represents a significant advancement in precision oncology.
  • Targeted inhibition of RET alterations offers a new therapeutic strategy.
  • The development pathway highlights the success of targeted drug development for RET-driven cancers.

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