21 July 2021 - - The US Food and Drug Administration has granted approval to US-based plasma provider Octapharma USA for Octagam 10% [Immune Globulin Intravenous (Human)], the first and only intravenous immunoglobulin to be indicated for the treatment of adult dermatomyositis, a rare immune-mediated inflammatory disease, the company said.

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 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 US, and is the largest study to evaluate intravenous immunoglobulin as a treatment option for dermatomyositis.

Dermatomyositis is a rare idiopathic autoimmune disorder of unknown cause affecting approximately 10 out of every m US 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.

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 /European League Against Rheumatism (EULAR) myositis response criteria.

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.

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 US Octapharma USA is located in Paramus, N.J.