ProDERM study results of octagam® 10% treatment in patients with dermatomyositis published in the New England Journal of Medicine
Octagam® 10% is efficacious and well-tolerated in adults with dermatomyositis
Octapharma announced today the results from the ProDERM study on the efficacy and safety of octagam® 10% [Immune Globulin Intravenous (Human)], in adult dermatomyositis (DM) patients have been published in the New England Journal of Medicine (Aggarwal R et al. “Efficacy and safety of intravenous immunoglobulin in dermatomyositis”.
Dermatomyositis is an immune-mediated myopathy characterised by chronic inflammation of the skin and muscles, leading to cutaneous rashes and progressive muscle weakness. DM is associated with increased morbidity and mortality due to muscle weakness and visceral involvement. Prior to the ProDERM study findings, no therapy had been approved in the US or Europe for the treatment of dermatomyositis based on randomized clinical trials.
The Progress in DERMatomyositis (ProDERM) study was the first large randomized clinical trial to investigate an intravenous immunoglobulin (IVIg) (octagam® 10%) in dermatomyositis. The results of the study demonstrated that octagam® 10% is an efficacious and well-tolerated treatment for adults with dermatomyositis. The study enrolled 95 adults from 36 sites in 10 countries and reported the following key findings:
The primary endpoint of the ProDERM study was met, with a significantly higher proportion of responders in the octagam® 10% group compared with the placebo group (79% vs. 44%; p <0.001) at Week 16 (end of the double-blind, placebo-controlled period).
Significantly more patients receiving octagam® 10% achieved a major or at least moderate improvement in the Total Improvement Score.
The efficacy of octagam® 10% was maintained through Week 40 (the end of the open-label extension period).
octagam® 10% was generally well tolerated. The safety and tolerability profile of IVIg was consistent with previously reported safety outcomes for IVIg administration.
“The lack of treatment options for dermatomyositis has hampered patient care to date,” said Rohit Aggarwal, MD, MS, Medical Director of the Arthritis and Autoimmunity Center at the University of Pittsburgh School of Medicine and chair of the ProDERM study Steering Committee. “The ProDERM study – and its publication in the New England Journal of Medicine – has been a clear turning point in the management of patients with this disease, with physicians now able to offer patients an effective treatment option with proven efficacy, safety and tolerability.” Wolfgang Frenzel, MD, Board Member and Head of Research and Development at Octapharma added, “We are very proud that octagam® 10% is the first product to demonstrate efficacy in a large, randomized controlled trial in dermatomyositis.”
“The ProDERM trial has given clinicians much more confidence in the efficacy and safety of octagam® 10% for adult DM patients,” said Octapharma USA President Flemming Nielsen. "The New England Journal of Medicine journal article is an exciting milestone for both providers and patients who previously relied on unapproved treatments for the disorder. We look forward to partnering with patient organizations and the medical community to develop educational and other support programs that will serve dermatomyositis patients.”
Early diagnosis and treatment are important for optimal management of patients with dermatomyositis, but diagnosis can be very challenging. “At Octapharma, we are committed to improving early detection and management of this rare disease,” said Olaf Walter, MD, MBA, Board Member and Head of International Business Units (IBUs) at Octapharma. “Our activities include the launch of a disease awareness website for healthcare practitioners (www.managedermatomyositis.com) and participation in congresses, such as the upcoming ACR (American College of Rheumatology) Convergence, taking place in November in Philadelphia, where the latest advances on this disorder are being shared.”
Following the results of the ProDERM study, octagam® 10% has received approval in the US for the “treatment of dermatomyositis in adults” and in Europe for “immunomodulation in adults with active dermatomyositis treated with immunosuppressive drugs including corticosteroids, or with intolerance or contra-indications to those drugs.”
About the ProDERM study
The ProDERM study (NCT02728752) was an international, multicenter, double-blind, randomized, placebo-controlled phase III clinical trial that investigated the efficacy, safety and tolerability of octagam® 10% in adults with dermatomyositis. In the initial 16-week placebo-controlled period, 95 patients from 36 sites in 10 countries were randomized to receive either octagam® 10% (2.0 g/kg) or placebo every four weeks. This was followed by an open-label extension period during which all patients received octagam® 10% for a further 24 weeks (excluding patients who had shown clinical worsening while receiving octagam® 10% in the first period). The primary endpoint was the proportion of patients who responded to treatment at Week 16.
Octagam® 10% is a ready to use, liquid preparation of highly purified human immunoglobulin for intravenous administration. octagam® 10% is approved for idiopathic thrombocytopenic purpura in the USA, EU and Canada. It is also approved for use in treatment of primary immunodeficiency, secondary immunodeficiencies and Guillain Barré syndrome in the EU and Canada, for dermatomyositis in the EU and the USA, and for chronic inflammatory demyelinating polyradiculoneuropathy (CIDP), multifocal motor neuropathy (MMN), and Kawasaki disease in the EU.
FOR THE US:
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.
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 10,000 people worldwide to support the treatment of patients in 118 countries with products across three therapeutic areas: immunotherapy, hematology, and critical care.
Octapharma has seven R&D sites and five state-of-the-art manufacturing facilities in Austria, France, Germany and Sweden, and operates more than 180 plasma donation centers across Europe and the US.
The company’s American subsidiary, Octapharma USA, is located in Paramus, N.J. Octapharma operates three state-of-the-art production sites licensed by the U.S. Food and Drug Administration (FDA), providing a high level of production flexibility.