On July 15, the U.S. Food & Drug Administration (FDA) approved a 10% solution of a human intravenous immunoglobulin (IVIG), Octagam 10%, to treat adults with dermatomyositis, a rare chronic systemic autoimmune disease with a characteristic skin rash and progressive proximal muscle weakness.1,2 This treatment was previously FDA approved to treat chronic immune thrombocytopenic purpura.3
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The current FDA approval was based on results of the international, multi-center study Progress in Dermatomyositis (ProDERM) study. Data from this phase 3, randomized, double-blind, placebo-controlled trial were presented during the 2021 virtual EULAR Congress, June 2–5, by Rohit Aggarwal, MD, MS, associate professor of medicine at the University of Pittsburgh, medical director of the Arthritis and Autoimmunity Center and co-director of the Myositis Center.4 The goal of the study (NCT02728752) was to evaluate the safety, tolerability and efficacy of IVIG in dermatomyositis patients.5
The study’s primary endpoint was the proportion of patients who responded to IVIG treatment compared with the proportion of patients who responded to placebo at week 16. Per the 2016 ACR/EULAR myositis response criteria, response was defined as a minimal improvement in total improvement score (TIS) of at least 20 points and no clinical worsening at two consecutive visits up to week 16.
The first part of the study lasted for 16 weeks. It was the double-blind, placebo-controlled period during which adult patients with definite or probable dermatomyositis were randomized 1:1 to receive either high-dose IVIG (2 g/kg of body weight) every four weeks or placebo.
In the second part of the study, patients treated with placebo and patients who did not have clinical worsening while receiving IVIG entered a 24-week, open-label extension period. These patients received 2 g/kg of body weight of IVIG every four weeks. Patients who were stable at week 28 could have their dose reduced to 1 g/kg of body weight every four weeks. Patients with clinical worsening at two consecutive visits between weeks 8 and 16 were switched to the alternate treatment arm.
A total of 95 adults with a mean age of 53 years were enrolled in ProDERM. The patients were predominantly white (92%) women (75%). Their baseline clinical characteristics, including medical history and prior dermatomyositis treatment, were well balanced between the IVIG treatment group (n=47) and the placebo group (n=48).
At week 16, the primary study endpoint was met, with 78.7% of IVIG-treated patients responding to treatment compared with 43.8% of placebo-treated patients. This difference of 34.9% was considered significant (95% confidence interval [CI] 16.70–53.24; P=0.0008).
After patients in the placebo group switched to IVIG treatment in the open-label extension period, these patients achieved a response rate at week 40 similar to the week 16 response of patients who had originally received IVIG treatment.
TIS was compared between patients who received IVIG (group 1) and those who received placebo, and then were crossed over to IVIG at week 16 (group 2). At three specific time points, the mean TIS scores were:
- At week 16, 48.4% for group 1 compared with 21.6% for group 2;
- At week 28, 54% for group 1 compared with 44.4% for group 2; and
- At week 40, 55.44% for group 1 compared with 51.07% for group 2.
The safety and tolerability profile of IVIG was consistent with previously reported safety outcomes with IVIG administration. No new safety signals were identified.
Michele B. Kaufman, PharmD, BCGP, is a freelance medical writer based in New York City and a pharmacist at New York Presbyterian Lower Manhattan Hospital.
- News release: FDA approves Octopharma’s Octagam 10% for adult dermatomyositis. Octapharma AG. 2021 Jul 20.
- Supplemental approval letter BL 125062/674: Immunoglobulin intravenous (human). U.S. Food & Drug Administration. 2021 Jul 15.
- Approval letter BL 125062/234: Octagam. U.S. Food & Drug Administration. 2014 Jul 11.
- Aggarwal R, Charles-Schoeman C, Schessl J, et al. A randomized, double-blind, placebo-controlled phase 3 trial of IVIG 10% in patients with dermatomyositis, the ProDERM study: Results on efficacy and safety [abstract OP0008]. Ann Rheum Dis. 2021;80:4–5.
- Octapharma AG. Study evaluating efficacy and safety of Octagam 10% in patients with dermatomyositis (idiopathic inflammatory myopathy [IIM]). ClinicalTrials.gov. 2021 Apr 21.