Zilucoplan: A Newly Approved Macrocyclic Peptide for Treatment of Anti-Acetylcholine Receptor Positive Myasthenia Gravis

• Type: Article in International Scientific Journal
• Date: 2024

Authors

Costa, L (Author) Other; Carla Fernandes (Author) FFUP

Journal

Title: Drugs and Drug Candidates

Vol. 416

Pages: 311-327

Publisher: MDPI

Other information

Authenticus ID: P-011-WYT

DOI: 10.3390/ddc3020018

Abstract (EN): <jats:p>Zilucoplan is a synthetic macrocyclic peptide approved by the Food and Drug Administration (FDA), in October 2023, for the treatment of generalized myasthenia gravis. It is considered as an orphan drug that causes the inhibition of terminal complement cascade activation with a dual mechanism of action preventing the formation of the membrane attack complex (MAC) and the destruction of the neuromuscular junction. This drug has been demonstrated to be able to treat the generalized myasthenia gravis without significant adverse effects, with good efficacy, safety, and tolerability profile. Zilucoplan is not only innovative and promising in the therapeutics of generalized myasthenia gravis, but it could also be beneficial for the treatment of other diseases as well as a model for synthesis of analogues to improve pharmacological profile.</jats:p>

Language: English

Type (Professor's evaluation): Scientific