Acthar Gel, a medication developed and marketed by Mallinckrodt Pharmaceuticals, is a compound that enhances the production of steroids in the body. It has been approved by the U.S. Food and Drug Administration (FDA) for 19 different indications, including as a treatment for acute MS flare-ups in adults who have frequent and recurring relapses.
Although previous clinical trials have shown that Acthar Gel accelerated patients’ recovery following an acute attack, no study has demonstrated the medication actually could change the natural course of the disease.
To explore that possibility, Mallinckrodt undertook an observational registry study (NCT02633033). The main goal of the study was to monitor the course of MS, document all relapses and the recovery process in a group of MS patients who had been treated with Acthar Gel for at least six months.
The primary goal of the study was to assess the effect of Acthar Gel on symptoms associated with MS flare-ups after two months of treatment, based on the MSIS-29v1 scale (a tool that measures the impact of MS in physical and psychological aspects from a patient’s perspective).
The study enrolled 148 adult patients (average age 47) at 55 clinical sites across the U.S. who had been living with MS for approximately 10 years.
More than half of the participants (60%) had a history of intolerance or poor response to treatment with high-dose corticosteroids, and 58% reported having had at least one flare-up in the two years before enrolling in the study.
Top-line data from the study showed that patients treated with Acthar Gel not only reported marked improvements in their symptoms after two months of therapy, but also had a sustained response to the medication that was still apparent six months after enrolling in the study.
Moreover, physicians monitoring the registry study participants also reported significant improvements in their physical symptoms, based on the Expanded Disability Status Scale (EDSS, a scale that measures the degree of patients’ disabilities).
Treatment adverse side effects were consistent with those reported in previous studies.
“Mallinckrodt is committed to generating data that helps clarify to the prescriber those patients appropriate for treatment with Acthar Gel to manage MS relapses,” Steven Romano, MD, executive vice president and chief scientific officer at Mallinckrodt, said in a press release.
“The registry evaluated the effect of Acthar Gel in prospectively defined patients who were treated with Acthar Gel following an acute relapse. In addition, we look forward to 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,” Romano said.
Of note, the OPTIONS trial is accessing the safety of Acthar Gel in relapsing-remitting MS (RRMS) patients who have not responded to high dose steroids.
Mallinckrodt plans to present the full data from the registry study at an upcoming medical meeting.