The US FDA approval of Octagam is based on positive data from the Phase 3 ProDERM Study in 95 patients at 36 sites across the world
Octapharma USA has obtained the US Food and Drug Administration (FDA) approval for Octagam 10% [Immune Globulin Intravenous] to treat adult dermatomyositis.
Dermatomyositis is a rare idiopathic autoimmune disorder of unknown cause, characterised by skin rashes, chronic muscle inflammation and progressive muscle weakness.
It is associated with complications including difficulty swallowing, aspiration pneumonia, breathing problems and calcium deposits on muscles, skin and connective tissues.
Octapharma said that its Octagam 10% is the first and only intravenous immunoglobulin (IVIg) indicated for the treatment of adult dermatomyositis.
Octapharma USA president Flemming Nielsen said: “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.
“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 FDA approval is based on the results of the Phase 3 ProDERM clinical trial that assessed the efficacy and safety of intravenous immunoglobulin (IVIg) in treating dermatomyositis.
The study enrolled 95 patients at 36 sites globally, including 17 sites in the US, and is said to be the largest study to evaluate intravenous immunoglobulin for dermatomyositis treatment.
In the Phase 3 trial, patients were randomised to receive either high-dose Octagam 10% (2g/kg) or placebo every four weeks for an initial period of 16 weeks.
The initial phase demonstrated that 78.7% of patients receiving Octagam 10% responded positively to treatment, compared to 43.8% of participants receiving placebo.
The initial phase was followed by a 24-week open-label extension phase, where patients were allowed to switch treatment if their disease worsened during the trial.
IVIg treatment showed statistically significant improvement compared to placebo, meeting the overall primary endpoint, secondary endpoints and all the subcomponents of Total Improvement Score.
Also, the safety and tolerability profile of the treatment was consistent with previously reported safety outcomes for IVIg administration, said the company.
ProDERM study steering committee member Rohit Aggarwal said: “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.
“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.”