FDA Approves Immune Globulin Intravenous Treatment for Adult Dermatomyositis

The FDA has approved the first and only intravenous immunoglobulin (IVIg) (Octagam, Octapharma USA) indicated for use in adults with dermatomyositis. A rare immune-mediated inflammatory disease, dermatomyositis is an idiopathic autoimmune disorder of unknown cause that affects the lives of approximately 10 out of every 1 million US residents.

The FDA approval was based on the results from the randomized, prospective, double-blind, placebo-controlled ProDERM phase 3 clinical trial, which was conducted among 95 patients across 19 international sites and 17 sites in the United States. This trial is the first to evaluate the long-term efficacy and safety of IVIg in adults with dermatomyositis and is the largest to assess IVIg as a treatment option for dermatomyositis.

“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, in a press release. “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.”

Patients with dermatomyositis commonly experience skin rashes, chronic muscle inflammation, and progressive muscle weakness. These symptoms most frequently affect adults in their late 40s to early 60s and children aged 5 to 15 years.

With these symptoms, complications can occur such as difficulty swallowing, aspiration pneumonia, breathing problems, and calcium deposits on muscles, skin, and connective tissues. In light of these complications, patients with dermatomyositis have more than a 3 times increased mortality risk in comparison with the general population of the same age.

“The FDA approval of Octagam® 10% as a safe and effective treatment for dermatomyositis in adults is exciting news for patients who previously relied on unapproved treatments,” said Octapharma US President Flemming Nielsen, in the press release. "Octapharma is committed to providing life-saving and life-enhancing therapies for patients with rare diseases. We look forward to partnering with patient organizations and the medical community to develop educational and other support programs that will serve dermatomyositis patients.”

The IVIg treatment is a liquid preparation that is also indicated for chronic immune thrombocytopenic purpura in adults, as well as for dermatomyositis in adults.

REFERENCE

FDA Approves Octapharma’s Octagam® 10% for Adult Dermatomyositis. Paramus, NJ: Octapharma USA; July 20, 2021. [email] Accessed July 21, 2021.