H.P. Acthar Gel, Acthar
Evidence Grade A — Regulatory approved. 19994 published studies. 185 registered clinical trials.
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Corticotropin (sold as Acthar Gel) is an injectable preparation of ACTH — a hormone that stimulates the adrenal glands to produce cortisol and other anti-inflammatory steroids. It carries approximately 19 labelled conditions including infantile spasms (a severe seizure disorder in babies), nephrotic syndrome, and multiple sclerosis relapses. It is also one of the most commercially controversial medications in the US due to extraordinary price increases over the past two decades.
19,994 published studies: 8317 human, 9241 animal, 1471 in-vitro, 2382 reviews
Corticotropin is marketed as H.P. Acthar Gel (currently ANI Pharmaceuticals). It carries approximately 19 FDA-labelled indications including infantile spasms (its strongest evidence base), nephrotic syndrome, multiple sclerosis relapses, and rheumatic disorders.
Acthar Gel has been at the centre of major pricing and legal controversies. The price rose from approximately $40 per vial in 2001 to over $40,000, driven by successive acquisitions and orphan-like positioning despite broad labelling. The former manufacturer Mallinckrodt agreed to a $260 million settlement over antitrust concerns. Clinically, the strongest evidence supports its use in infantile spasms, where it is considered a first-line treatment. For most other indications, debate continues over whether it offers meaningful advantages over far less expensive oral corticosteroids.
Corticotropin stimulates the adrenal glands to produce cortisol, the body's primary anti-inflammatory hormone, and other adrenal steroids. This is its traditional mechanism. However, corticotropin also activates melanocortin receptors beyond the adrenal glands, particularly on immune cells, which may produce anti-inflammatory and immunomodulatory effects independent of cortisol production. This additional mechanism has been proposed as a reason why Acthar Gel may have effects beyond what oral corticosteroids alone provide, though this remains debated.
The strongest evidence for corticotropin is in infantile spasms, where it is considered a first-line treatment. For most of its other labelled conditions, the evidence is weak by modern standards, and there is ongoing debate about whether it offers meaningful advantages over far less expensive oral corticosteroids. The drug's commercial history has overshadowed its clinical profile. The price rose from approximately $40 per vial in 2001 to over $38,000, driven by successive corporate acquisitions. The manufacturer at the time, Mallinckrodt, agreed to a major antitrust settlement over allegations of illegally acquiring a potential competing product. Medicare spending on Acthar reached billions of dollars. Proponents argue that ACTH has immunomodulatory effects beyond what oral steroids provide through additional melanocortin receptor activation, but this claim remains debated.
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