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- Giuseppe Tringali and Pierluigi Navarra.
- Institute of Pharmacology, Fondazione Policlinico Universitario A. Gemelli IRCCS, Roma - Università Cattolica del Sacro Cuore, Largo F. Vito 1, 00168 Rome, Italy. Electronic address: giuseppe.tringali@unicatt.it.
- Peptides. 2019 Jun 1; 116: 16-21.
AbstractCalcitonin gene-related peptide (CGRP) is a peptide neurotransmitter with potent vasodilating properties. CGRP is believed to play a primary role in the pathogenesis of migraine. As such, CGRP and its receptors are obvious druggable targets for novel anti-migraine agents. While the development of small-molecule CGRP receptor antagonists started first, none of these agents is yet available in clinical practice. Conversely, both anti-CGRP and anti-CGRP receptor monoclonal antibodies (mABs) completed clinical development, and the first representatives of these 2 classes are available on the market. MABs are approved for prevention of migraine attacks in chronic or episodic migraine, involving long-term treatments. In light of the physiological role exerted by CGRP in the regulation of vascular tone, the potential risks of a long-term inhibition of CGRP functions raised diffuse concerns. These concerns were correctly addressed by the anti-CGRP receptor mABs erenumab with a 5-year open-label clinical trial; however, this study is currently ongoing and results are not yet available, leaving some uncertainty on the profile of erenumab long-term safety. Similar concerns can be raised with direct anti-CGRP mABs, which entrap the peptide preventing receptor activation. However, evidence exists that plasma CGRP is detectable in patients chronically treated with anti-CGRP mABs. Assuming that plasma CGRP is an indirect marker of peptide levels at the vascular receptor sites, such residual CGRP would maintain a physiological level of receptor stimulation, in spite of a well-established anti-migraine activity of the mABs. This might represent a potential advantage in the safety profile of anti-CGRP mABs, but it needs to be confirmed and expanded with data on free plasma CGRP.Copyright © 2019 Elsevier Inc. All rights reserved.
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