Acthar Gel is a repository corticotropin injection that is distributed by Mallinckrodt Pharmaceuticals. It is approved by the U.S. Food and Drug Administration (FDA) for the treatment of 19 conditions, including pulmonary sarcoidosis.
Acthar Gel was first approved by the FDA in 1952 based on case reports. Currently, it is administered as an injection under the skin (subcutaneously) or into the muscle tissue (intramuscularly). It is an alternative treatment for patients who do not tolerate or respond to prednisone (a corticosteroid), the first-line treatment for sarcoidosis.
How does Acthar Gel work?
Acthar Gel is a highly purified preparation of adrenocorticotropin hormone (ACTH) obtained from pituitary glands isolated from pigs, which is mixed with a gel and injected subcutaneously or intramuscularly so that it is released slowly into the bloodstream.
The ACTH hormone in Acthar Gel binds to melanocortin receptors (MCRs) in the adrenal glands on the kidneys and stimulates the production of natural corticosteroid hormones such as cortisol. These natural corticosteroids will then suppress the inflammatory and immune processes that are responsible for sarcoidosis.
Acthar Gel in clinical trials
A randomized, double-blind Phase 4 clinical trial (NCT02188017) in chronic pulmonary sarcoidosis patients who previously had received prednisone treatment, assessed whether Acthar Gel can reduce the requirement for prednisone. The study compared two different doses of Acthar Gel, 40 and 80 units, twice a week. Sixteen patients completed the 24-week study.
The results were published in the journal Lung and showed that Acthar Gel treatment significantly reduced the prednisone dose required within seven weeks of treatment and this persisted until the end of the study. Moreover, the diffusing capacity of the lungs for carbon monoxide (DLCO), which is part of a lung function test, and the general health status of the patients improved significantly during the study despite the withdrawal of prednisone. There was no significant difference in therapy response between the two doses.
A retrospective pilot study (NCT02356445) followed the clinical outcomes of Acthar Gel therapy in 47 patients with advanced sarcoidosis who were taking corticosteroids and in whom the disease was affecting one or more organs including the lungs, skin, eyes, or brain and spinal cord. All patients received 80 units of Acthar gel as a subcutaneous or intramuscular injection, twice a week. Eighteen patients discontinued therapy: four patients because of the cost of the treatment; two patients died; 11 patients experienced toxicity); and one patient was noncompliant. Meanwhile, 29 patients received Acthar gel therapy for at least three months.
Results of that study were published in the journal Respiratory Medicine and showed that 11 of the 29 patients reported improvements in one or more affected organs. The corticosteroid dose in eight of these 11 patients was lowered by at least 50%. Sixteen of the 29 patients did not show improvements, but their disease remained stable, whereas it worsened in the remaining two patients.
A randomized, double-blind Phase 4 clinical trial (NCT03320070) is recruiting 100 pulmonary sarcoidosis patients who are receiving prednisone treatment, in multiple centers around the U.S. to assess the effectiveness and safety of Acthar Gel. Patients will receive either 80 units of Acthar Gel or a placebo twice a week for 24 weeks. They will be tested for improvements in lung function and quality of life, undergo high-resolution CT scans to evaluate pulmonary or extrapulmonary disease, and be assessed in terms of the prednisone dose they require, all of which will be recorded as a sarcoidosis treatment score (STS). This study is expected to be completed in May 2020.
Two more clinical trials are designed to assess the effect of Acthar Gel in patients with sarcoidosis affecting the central nervous system (neurosarcoidosis). One trial (NCT02298491) recruited four participants in Maryland to study whether treatment with Acthar Gel can be effective in improving clinical and radiographic abnormalities and to assess any improvements in patients’ quality of life. The trial was completed in July and results are forthcoming.
The second trial (NCT02920710) will study whether Acthar Gel can be an effective alternative treatment for patients with neurosarcoidosis. It aims to recruit 20 participants in Ohio, U.S. and is expected to be completed in December 2020. The primary outcome is to estimate the proportion of patients with clinically significant improvements after treatment with Acthar Gel for 12 weeks that would allow the tapering of corticosteroid treatment.
Mallinckrodt reported the results of a large retrospective study about the effectiveness of Acthar Gel in treating symptomatic sarcoidosis in the journal Therapeutic Advances in Respiratory Diseases. This study included 302 patients. Most patients had extrapulmonary involvement, primarily in the skin (28%), joints (25%), heart (22%), and eyes (22%), and 34% of patients had two or more organs involved. The mean duration of the treatment with Acthar Gel was 32.5 weeks and 61.6% of patients continued the therapy for more than six months.
Results showed that patients treated with a higher starting dose of Acthar Gel required the therapy for a shorter duration than those treated with a lower starting dose. Moreover, treatment with Acthar Gel reduced the percentage of patients using corticosteroids from 61.3% before initiation of Acthar Gel to 12.9% after Acthar Gel therapy for at least three months. The average daily corticosteroid dose required decreased from 18.2 mg to 9.9 mg, with the number of patients requiring a low dose of prednisone (less than 10 mg/day) increasing from 21% before Acthar Gel treatment to 47.3% three months after Acthar Gel treatment. The overall Acthar Gel treatment response was 95%, with 73% of patients reporting improvements in overall symptoms, 38% showing lung function improvement and 33% showing decreased inflammation.
Acthar Gel’s side effects are similar to those of corticosteroids, the most common being high blood sugar, high blood pressure, behavior and mood changes, and changes in appetite and weight.
The use of Acthar Gel can increase the risk of infections because it suppresses the immune response.
Last updated: Nov. 30, 2019
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