Therapy Areas: Cardiovascular
Octapharma's Octagam 10% Receives Seven Years of Market Exclusivity for Adult Dermatomyositis with FDA Orphan Drug Approval
30 August 2021 - - The US Food and Drug Administration Office of Orphan Products Development (OOPD) has awarded seven years of marketing exclusivity for Octagam 10% [Immune Globulin Intravenous (Human)], the first and only intravenous immunoglobulin to be indicated for the treatment of adult dermatomyositis, US-based human protein manufacturer Octapharma USA said.

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.

The FDA grants orphan drug status to novel therapies that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 U.S residents.

After market approval for the orphan designation, the FDA/OOPD will award seven years of market exclusivity.

The FDA approval is based on the results of ProDERM ( Identifier: NCT02728752), a pivotal randomized clinical trial and the first study to evaluate the long term efficacy and safety of 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 US, and is the largest study to evaluate intravenous immunoglobulin as a treatment option for DM.

Octagam 10% [Immune Globulin Intravenous (Human)] is an immune globulin intravenous (human) liquid preparation indicated for the treatment of chronic immune thrombocytopenic purpura in adults; and dermatomyositis in adults.

Thrombosis may occur with immune globulin intravenous 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) 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.