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Review
. 2023 Oct;43(10):739-761.
doi: 10.1007/s40261-023-01303-5. Epub 2023 Oct 4.

Acthar® Gel Treatment for Patients with Autoimmune and Inflammatory Diseases: An Historical Perspective and Characterization of Clinical Evidence

Jeffrey Kaplan  1 , Anca Askanase  2 , David Chu  3 , Abdul Abdellatif  4 , Dhiman Basu  5 , Mehdi Mirsaeidi  6
Affiliations
Review

Acthar® Gel Treatment for Patients with Autoimmune and Inflammatory Diseases: An Historical Perspective and Characterization of Clinical Evidence

Jeffrey Kaplan et al. Clin Drug Investig. 2023 Oct.

Abstract

Acthar® Gel (repository corticotropin injection) is a naturally sourced complex mixture of adrenocorticotropic hormone analogs and other pituitary peptides that is believed to have both steroidogenic and nonsteroidogenic immunomodulatory effects via activation of melanocortin receptors in various cells throughout the body. Since 1952, Acthar has been approved by the US Food and Drug Administration to treat a variety of autoimmune and inflammatory diseases. Since 2014, Mallinckrodt Pharmaceuticals has conducted a large number of preclinical, clinical, and real-world-evidence studies of Acthar for the treatment of rheumatoid arthritis, systemic lupus erythematosus, dermatomyositis and polymyositis, multiple sclerosis relapse, ophthalmic disorders, sarcoidosis, and nephrotic syndrome. To date, Acthar has been the subject of more than 500 publications, many of which demonstrate the safety and efficacy of Acthar in patients with inflammatory diseases for whom standard treatments were ineffective or intolerable. Here, we review the history of Acthar and the findings of studies that have investigated the mechanism of action, safety, efficacy, and real-world effectiveness of Acthar for the treatment of inflammatory diseases.

Plain language summary

Acthar® Gel is an anti-inflammatory drug that directly affects the immune system in a manner that differs from other anti-inflammatory drugs, such as corticosteroids. Since 1952, Acthar has been approved by the U.S. Food and Drug Administration to treat a variety of diseases involving inflammation. The commercial rights to produce Acthar have changed hands several times over the years, beginning with Armour Pharmaceuticals and most recently ending with Mallinckrodt Pharmaceuticals in 2014. Since then, Mallinckrodt has conducted multiple studies in animals to demonstrate the function of Acthar compared with other anti-inflammatory drugs. Further, several clinical trials in humans and studies of hospital or clinical practice records have confirmed the safety and effectiveness of Acthar as a treatment for many inflammatory diseases. INFOGRAPHIC: A podcast discussion by the authors on Acthar® Gel treatment for patients with autoimmune and inflammatory diseases, including their own personal reflections and experiences with Acthar® Gel. For a transcript of the podcast see the electronic supplementary material. (MP4 177883 kb).

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Conflict of interest statement

Anca Askanase has been an investigator or consultant for AbbVie, Amgen, AstraZeneca, Aurinia, Bristol Myers Squibb, Celgene, Eli Lilly, Idorsia, Janssen, Genentech, GSK, Mallinckrodt, Pfizer, and UCB. Abdul Abdellatif has been a consultant and paid speaker for Horizon Therapeutics. David Chu has received consulting fees or a research grant from Aldeyra, Bausch and Lomb, Kriya, Mallinckrodt, and Santen. Dhiman Basu and Mehdi Mirsaeidi have no disclosures to report. Jeffrey Kaplan has received fees as a speaker or consultant for Alexion, Allergan, Amgen, Biogen, Biohaven, Bristol Myers Squibb, EMD Serono, Genentech, Genzyme, Horizon, Impel, Janssen, Lundbeck, Mallinckrodt, Merck, Novartis, and Teva.

Figures

Fig. 1
Fig. 1
Timeline of key events regarding Acthar. DESI Drug Efficacy Study Implementation, FDA US Food and Drug Administration, IS infantile spasms, MS multiple sclerosis
See this image and copyright information in PMC

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References

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