Acthar Gel Quite Effective at Resolving Relapses as Alternative to Corticosteroids, Study Reports

Acthar Gel (repository corticotropin injection) is more effective for treating flares in multiple sclerosis (MS) than other alternatives, including intravenous immunoglobulin or plasmapheresis, a claims-based study from Mallinckrodt Pharmaceuticals reports.

The study “Treatment Effectiveness for Resolution of Multiple Sclerosis Relapse in a US Health Plan Population” was published in the journal Neurology and Therapy.

Acthar Gel, developed and marketed by Mallinckrodt Pharmaceuticals, is approved by the U.S. Food and Drug Administration (FDA) for 19 different indications, including the treatment of acute MS flare-ups in adults who have frequent and recurring relapses.

The compound consists of a gel preparation of adrenocorticotropic hormone (ACTH), which is injected under the skin (subcutaneously) or into the muscle (intramuscularly). It works by enhancing the production of steroids in the body to reduce inflammation and more rapidly resolve a flare.

Acthar Gel is often used as a treatment alternative (or second-line) in patients who cannot tolerate the side effects of, or have responded poorly to, high-dose corticosteroids (the standard treatment).

Clinical trials have demonstrated that Acthar Gel speeds patients’ recovery from a MS relapse compared to placebo, but no study has shown that it changes the disease’s natural course.

This newly published retrospective study, conducted by Mallinckrodt researchers, evaluated the rate of MS relapses, frequency of treatments used, and their effectiveness (relapse resolution rates) in patients using standard corticosteroid therapy or other alternatives, including Acthar Gel, intravenous immunoglobulin, and plasmapheresis.

Researchers reviewed data covering 9,574 adults with MS flares between 2008 and 2015, using administrative claims data from the insurance company Humana. A total of 25,162 relapse episodes were identified. The mean follow-up time per patient was 2.7 years.

The majority of patients had less than two flares a year, while 26% experienced two or more acute attacks annually. Over the study period, about one third (36.9%) did not recover from one or more flares and required alternative relapse treatments.

Corticosteroids were the first treatment used in 90.4% of the initial relapses — 51.8% with oral corticosteroids, and 38.6% with injections of methylprednisolone (sold as Solu-Medrol). Alternatives used included intravenous immunoglobulin (6.0% of relapses), Acthar Gel (2.2%), and plasmapheresis (1.5%).

The proportion of patients achieving relapse resolution with their first treatment varied considerably among these medications. Treatment was considered effective if no subsequent attacks followed a relapse.

Oral corticosteroids led to a successful relapse resolution in 90.5% of patients (5,710 people), which agrees with prior studies and further supports these therapies being “generally effective,” the researchers wrote. In comparison, methylprednisolone was less effective, treating less than half of the cases (47.8%; 3425 patients).

Noticeably, the study showed that Acthar Gel was the most effective treatment alternative to corticosteroids, resolving relapses in 96.9% of the cases (195 patients), compared to a lower rate of resolutions for intravenous immunoglobulin (43.9%, 171 patients), and plasmapheresis (50.7%, 73 patients).

These data demonstrate Acthar Gel’s “effectiveness in appropriate patients,” the researchers wrote. More studies regarding the treatment’s safety, however, are needed.

“The management of MS relapse is an ongoing challenge, in particular for those patients who need additional treatment options after first-line agents such as corticosteroids,” George Wan, PhD, Mallinckrodt’s vice president and global head of health economics and outcomes research, said in a press release.

“We are committed to working toward a better understanding of the potential clinical and health economic outcomes associated with Acthar Gel and other late-line treatments for MS relapse to help guide clinical practice,” Wan added. “In addition, we look forward to the results of Mallinckrodt’s ongoing, randomized, double-blind, placebo-controlled OPTIONS study, which we anticipate will provide data on some of the more difficult-to-treat MS patients in the future.”

Mallinckrodt’s OPTIONS study (NCT03126760) is evaluating the efficacy and safety of multiple doses of Acthar Gel in adults with relapsing-remitting MS (RRMS) who have not responded to high-dose steroids. The trial is recruiting patients at 30 sites across the U.S.; contact and location information is available using this link.