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FDA approved Octagam 10% : First intravenous immunoglobulin for adult dermatomyositis!
FDA approved Octagam 10% : First intravenous immunoglobulin for adult dermatomyositis! New medicine for dermatomyositis! The US FDA approved Octagam 10%: the first intravenous immunoglobulin (IVIg) for the treatment of adult dermatomyositis!
Octapharma USA recently announced that the U.S. Food and Drug Administration (FDA) has approved Octaam 10% (intravenous immunoglobulin [human]), which is the first and only drug used to treat adult dermatomyositis (dermatomyositis, DM) intravenous immunoglobulin (IVIg).
Dermatomyositis is a rare immune-mediated inflammatory disease. Octagam 10% is an immunoglobulin [human] intravenous injection solution that has been approved in the United States for the treatment of chronic immune thrombocytopenic purpura (ITP) in adults.
The FDA approved Octagam to treat dermatomyositis based on the results of the Phase 3 ProDERM clinical trial (ClinicalTrials.gov registration number: NCT02728752). This is a pivotal randomized clinical trial and the first study to evaluate the long-term efficacy and safety of IVIg in the treatment of adult dermatomyositis.
This prospective, double-blind, placebo-controlled Phase 3 clinical trial enrolled 95 patients in 36 clinical centers around the world (including 17 in the United States) and is the largest study evaluating IVIg as a treatment option for dermatomyositis.
Rohit Aggarwal, member of the PrimDRM Research Steering Committee and medical director of the Arthritis and Autoimmune Center at the University of Pittsburgh School of Medicine, said: “The ProDERM research will have a significant impact on clinical practice because IVIg will become an important treatment option for patients with dermatomyositis. This research It gives clinicians more confidence in the efficacy and safety of IVIg, and provides valuable information about which types of patients are most suitable for treatment.”
Dermatomyositis is a rare idiopathic autoimmune disease of unknown cause, affecting approximately 10 people per million American residents. Patients with dermatomyositis usually suffer from skin rash, chronic muscle inflammation, and progressive muscle weakness. The disease usually affects adults in their 40s to 60s and children from 5 to 15 years old. Complications of dermatomyositis include dysphagia, aspiration pneumonia, breathing problems, and calcium deposits in muscles, skin, and connective tissues. Compared with the matched general population, the mortality rate of patients with dermatomyositis is more than three times higher.
Flemming Nielsen, President of Octapharma USA, said: “The FDA’s approval of Octaam® 10% as a safe and effective treatment for adult dermatomyositis is exciting news for patients who have previously relied on unapproved treatments. Octaharma is committed to providing life-saving and life-enhancing therapies for patients with rare diseases. We look forward to working with patient organizations and the medical community to develop education and other support programs to provide services to patients with dermatomyositis.”
The ProDerm clinical trial included an initial 16-week, double-blind, placebo-controlled period, where patients were randomized to receive high-dose Octagam 10% (2g/kg) or placebo once every 4 weeks. The initial treatment period is followed by a 24-week open label expansion period. If the patient’s condition deteriorates during the trial, they are allowed to change the treatment plan. In the study, the 2016 American College of Rheumatology (ACR)/European Union Against Rheumatism (EULAR) Myositis Response Standard was used to measure patient response to treatment.
The results showed that in the first 16-week double-blind treatment period, 78.7% of patients who received Octagam responded positively to treatment, while 43.8% of patients who received placebo responded positively to treatment.
After switching to IVIg during the expansion period, the response rate of the placebo group at week 40 was similar to the response rate of the Octagam 10% group at week 16 (minimum improvement was 70%).
Consistent with the overall primary endpoint, the secondary endpoints (including all sub-items of the overall improvement scale [TIS] except muscle enzymes, dermatomyositis disease area and severity index [CDASI]), compared with placebo , IVIg treatment also showed a statistically significant improvement.
In the study, the safety and tolerability of IVIg are consistent with the previously reported safety results of IVIg treatment.
(source:internet, reference only)