The Food and Drug Administration (FDA) has approved Octagam® 10% (immune globulin intravenous [human]) for the treatment of adult dermatomyositis, a rare immune-mediated inflammatory disease.
The approval was based on data from the prospective, double-blind, randomized, placebo-controlled phase 3 ProDERM study (ClinicalTrials.gov Identifier: NCT02728752), which evaluated the efficacy and safety of Octagam 10% in 95 adults with dermatomyositis. Patients were randomly assigned to receive either Octagam 10% or placebo intravenously (IV) every 4 weeks during the initial 16 week period, followed by a 24-week open-label extension phase during which all patients received Octagam 10% every 4 weeks. The primary endpoint was the proportion of responders at week 16, defined as a patient with an increase of at least 20 points on the Total Improvement Score (TIS).
Findings demonstrated that 78.7% of patients treated with Octagam 10% were responders at week 16 compared with 43.8% of those who received placebo (difference, 35.0%; 95% CI, 16.7-53.2; P =.0008). The median time to response was 35 days in the Octagam 10% group.
Compared with placebo, a greater proportion of patients in the Octagam 10% group had at least moderate improvement, defined as an increase of at least 40 points on the TIS, and major improvement, defined as an increase of at least 60 points on the TIS. The efficacy of Octagam 10% was also supported by improvement in the Cutaneous Dermatomyositis Disease Area and Severity Index total activity score. Among 46 patients who switched to Octagam 10% from placebo in the open-label extension phase, 69.6% were responders at week 40.
“The ProDERM study will have a significant impact on clinical practice because IVIg is likely to become an important treatment option for patients with dermatomyositis,” said Rohit Aggarwal, MD, MS, Medical Director of the Arthritis and Autoimmunity Center at the University of Pittsburgh School of Medicine and a member of the ProDERM study steering committee. “The study gives clinicians much more confidence in the efficacy and safety of intravenous immunoglobulin and provides valuable information about what type of patient is best suited for the treatment.”
Octagam 10% is already approved for the treatment of chronic immune thrombocytopenia purpura. The product is available as 2g, 5g, 10g, 20g or 30g single-use bottles.