Zilucoplan: First Approval

Shirley, Matt

doi:10.1007/s40265-023-01977-3

Cited by 7 publications

(4 citation statements)

References 9 publications

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“…Another drug approved by the FDA in 2023 for treating generalized myasthenia gravis is zilucoplan . Zilucoplan is a small, synthetic macrocyclic peptide inhibitor of the complement component 5 (Shirley 2023b ), by preventing the cleavage of C5 into C5a and C5b and also by inhibiting the binding of C5b to C6 (Tang et al 2023 ). Thus, zilucoplan has been suggested to have a dual mechanism of action preventing complement activation (Tang et al 2023 ).…”

Section: Neurologymentioning

confidence: 99%

“…Thus, zilucoplan has been suggested to have a dual mechanism of action preventing complement activation (Tang et al 2023 ). Zilucoplan has been approved for use in adult patients who are positive for anti-AChR antibodies (Shirley 2023b ) and granted orphan drug status (Menon and Bril 2022 ). The most common AE are bruising and pain at the injection site, headache, diarrhea, and exacerbation of the disease (Shirley 2023b ).…”

Section: Neurologymentioning

confidence: 99%

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A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023

Kayki-Mutlu,

Aksoyalp,

Wojnowski

et al. 2024

Naunyn-Schmiedeberg's Arch Pharmacol

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With 54 new drugs and seven cellular and gene therapy products, the approvals by the US Food and Drug Administration (FDA) recovered 2023 from the 2022 dent back to the levels of 2020–2021. As in previous years of this annual review, we assign these new drugs to one of three levels of innovation: first drug against a condition (“first-in-indication”), first drug using a novel molecular mechanism (“first-in-class”), and “next-in-class,” i.e., a drug using an already exploited molecular mechanism. We identify four (7%) “first-in-indication,” 22 (36%) “first-in-class,” and 35 (57%) “next-in-class” drugs. By treatment area, rare diseases (54%) and cancer drugs (23%) were once again the most prevalent (and partly overlapping) therapeutic areas. Other continuing trends were the use of accelerated regulatory approval pathways and the reliance on biopharmaceuticals (biologics). 2023 marks the approval of a first therapy based on CRISPR/Cas9 gene editing.

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Section: Neurologymentioning

confidence: 99%

Section: Neurologymentioning

confidence: 99%

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023

Kayki-Mutlu,

Aksoyalp,

Wojnowski

et al. 2024

Naunyn-Schmiedeberg's Arch Pharmacol

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“…The newly approved cyclic peptide was zilucoplan (Figure 1), a macrocyclic peptide used for treatment of generalized myasthenia gravis [25,26].…”

Section: Introductionmentioning

confidence: 99%

“…The newly approved cyclic peptide was zilucoplan (Figure 1), a macrocyclic peptide used for treatment of generalized myasthenia gravis [25,26]. Zilucoplan (Table 1), also designated as RA101495, is the active principle of Zilbrysq ® , commercialized by UCB Pharma S.A..…”

Section: Introductionmentioning

confidence: 99%

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

Costa,

Fernandes

2024

DDC

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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.

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Complement‐targeted therapeutics: Are we there yet, or just getting started?

Ricklin

2024

Eur J Immunol

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Therapeutic interventions in the complement system, a key immune‐inflammatory mediator and contributor to a broad range of clinical conditions, have long been considered important yet challenging or even unfeasible to achieve. Almost 20 years ago, a spark was lit demonstrating the clinical and commercial viability of complement‐targeted therapies. Since then, the field has experienced an impressive expansion of targeted indications and available treatment modalities. Currently, a dozen distinct complement‐specific therapeutics covering several intervention points are available in the clinic, benefiting patients suffering from eight disorders, not counting numerous clinical trials and off‐label uses. Observing this rapid rise of complement‐targeted therapy from obscurity to mainstream with amazement, one might ask whether the peak of this development has now been reached or whether the field will continue marching on to new heights. This review looks at the milestones of complement drug discovery and development achieved so far, surveys the currently approved drug entities and indications, and ventures a glimpse into the future advancements yet to come.

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Zilucoplan: First Approval

Cited by 7 publications

References 9 publications

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023

A year in pharmacology: new drugs approved by the US Food and Drug Administration in 2023

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

Complement‐targeted therapeutics: Are we there yet, or just getting started?

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