FDA approval based on positive results of international, multi-center ProDERM study
PARAMUS, N.J. – The U.S. Food and Drug Administration (FDA) has granted approval to Octapharma USA for Octagam® 10% [Immune Globulin Intravenous (Human)], the first and only intravenous immunoglobulin (IVIg) to be indicated for the treatment of adult dermatomyositis, a rare immune-mediated inflammatory disease.
The FDA approval is based on the results of ProDERM (ClinicalTrials.gov Identifier: NCT02728752), a pivotal randomized clinical trial and the first study to evaluate the long term efficacy and safety of intravenous immunoglobulin (IVIg) for adults with dermatomyositis. The prospective, double-blind, placebo-controlled Phase III clinical trial enrolled 95 patients at 36 sites globally, including 17 sites in the U.S., and is the largest study to evaluate intravenous immunoglobulin 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. “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.”
Dermatomyositis is a rare idiopathic autoimmune disorder of unknown cause affecting approximately 10 out of every million U.S. residents. Patients commonly suffer from skin rashes, chronic muscle inflammation and progressive muscle weakness, usually affecting adults in their late 40s to early 60s and children between the ages of 5 and 15. Complications include difficulty swallowing, aspiration pneumonia, breathing problems and calcium deposits on muscles, skin and connective tissues. DM patients have more than a three-fold higher risk of mortality compared with the matched general population.1, 2, 3, 4
“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 USA President Flemming Nielsen. “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 ProDerm clinical trial included an initial 16-week, double-blind, placebo-controlled period where patients were randomized to receive either high-dose Octagam® 10% (2g/kg) or placebo every four weeks. The initial treatment period was followed by a 24-week open label extension phase. Patients were allowed to switch treatment if they deteriorated during the trial. Patient response to treatment was measured using the 2016 American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) myositis response criteria.5
During the initial 16-week phase, 78.7% of patients receiving Octagam® 10% responded positively to treatment as compared to 43.8% of those receiving placebo. After switching to IVIg in the extension period, the placebo group attained a similar response rate at Week 40 as did the Octagam® 10% treated patients at Week 16 (approximately 70% for minimal improvement). In line with the overall primary endpoint, secondary endpoints, including all of the subcomponents of Total Improvement Score except muscle enzyme, and Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), also showed statistically significant improvement under IVIg treatment compared to placebo. The safety and tolerability profile of IVIg was consistent with previously reported safety outcomes for IVIg administration.
About Octagam® 10%
Octagam® 10% [Immune Globulin Intravenous (Human)] is an immune globulin intravenous (human) liquid preparation indicated for the treatment of:
? Chronic immune thrombocytopenic purpura (ITP) in adults
? Dermatomyositis (DM) in adults
THROMBOSIS, RENAL DYSFUNCTION AND ACUTE RENAL FAILURE
Thrombosis may occur with immune globulin intravenous (IGIV) products, including Octagam® 10%. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors. Renal dysfunction, acute renal failure, osmotic nephropathy, and death may occur with the administration of immune globulin intravenous (Human) (IGIV) products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. Octagam® 10% does not contain sucrose. For patients at risk of thrombosis, renal dysfunction or renal failure, administer Octagam® 10% at the minimum infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity.
For full prescribing information, including complete boxed warning, please click here.
To learn more about Octagam 10% for the treatment of dermatomyositis, please click here.
About the Octapharma Group
Headquartered in Lachen, Switzerland, Octapharma is one of the largest human protein manufacturers in the world, developing and producing human proteins from human plasma and human cell lines.
Octapharma employs more than 9,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: Hematology, Immunotherapy and Critical Care.
Octapharma has seven research and development sites and six state-of-the-art manufacturing facilities in Austria, France, Germany, Mexico and Sweden, and operates more than 160 plasma donation centers across Europe and the U.S. Octapharma USA is located in Paramus, N.J. For more information, please visit http://www.
1 – Dermatomyositis; U.S. Department of Health & Human Services, National Institutes of Health, National Center for Translational Sciences, website: https:/
2 – Dermatomyositis; Mayo Clinic, website: https:/
3 – Cordeiro AC, Isenberg DA. Treatment of inflammatory myopathies. Postgrad Med J 2006; 82:417-24.
4 – D’Silva, K. M. et al. Persistent premature mortality gap in dermatomyositis and polymyositis: a United Kingdom general population-based cohort study. Rheumatology 2020; DOI: 10.1093.
5 – Aggarwal R, et al. 2016 American College of Rheumatology/European League Against Rheumatism Criteria for Minimal, Moderate, and Major Clinical Response in Adult Dermatomyositis and Polymyositis. Arthritis Rheumatol 2017; 69:898-910.