Blocking the CGRP Pathway for Acute and Preventive Treatment of Migraine: The Evolution of Success

J Med Chem. 2020 Jul 9;63(13):6600-6623. doi: 10.1021/acs.jmedchem.9b01810. Epub 2020 Feb 20.

Abstract

The pivotal role of calcitonin gene-related peptide (CGRP) in migraine pathophysiology was identified over 30 years ago, but the successful clinical development of targeted therapies has only recently been realized. This Perspective traces the decades long evolution of medicinal chemistry required to advance small molecule CGRP receptor antagonists, also called gepants, including the current clinical agents rimegepant, vazegepant, ubrogepant, and atogepant. Providing clinically effective blockade of CGRP signaling required surmounting multiple challenging hurdles, including defeating a sizable ligand with subnanomolar affinity for its receptor, designing antagonists with an extended confirmation and multiple pharmacophores while retaining solubility and oral bioavailability, and achieving circulating free plasma levels that provided near maximal CGRP receptor coverage. The clinical efficacy of oral and intranasal gepants and the injectable CGRP monoclonal antibodies (mAbs) are described, as are recent synthetic developments that have benefited from new structural biology data. The first oral gepant was recently approved and heralds a new era in the treatment of migraine.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Animals
  • Calcitonin Gene-Related Peptide / metabolism*
  • Calcitonin Gene-Related Peptide Receptor Antagonists / pharmacology
  • Calcitonin Gene-Related Peptide Receptor Antagonists / therapeutic use
  • Humans
  • Migraine Disorders / drug therapy*
  • Migraine Disorders / metabolism
  • Migraine Disorders / pathology
  • Migraine Disorders / prevention & control*
  • Receptors, Calcitonin Gene-Related Peptide / metabolism
  • Signal Transduction / drug effects*

Substances

  • Calcitonin Gene-Related Peptide Receptor Antagonists
  • Receptors, Calcitonin Gene-Related Peptide
  • Calcitonin Gene-Related Peptide