FDA grants seven years of marketing exclusivity for Octapharma’s Octagam 10%

Octapharma USA today announced the U.S. Food and Drug Administration (FDA) 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 (IVIg) to be indicated for the treatment of adult dermatomyositis (DM).

The FDA approval of orphan drug status for Octagam^® 10% to treat adults with DM should encourage patients to ask their physicians about the therapy because they no longer have to rely on treatments that have not been specifically evaluated by the FDA for DM. We expect that Octagam^® 10% may become the first treatment option for the vast majority of adults with DM given the outstanding results from the ProDERM clinical trial."

Flemming Nielsen, President , Octapharma-USA

Dermatomyositis is a rare idiopathic autoimmune disorder of unknown cause affecting approximately 10 out of every million U.S. 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.^{1, 2, 3, 4}

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. Aftermarket 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 (ClinicalTrials.gov 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 U.S., and is the largest study to evaluate intravenous immunoglobulin as a treatment option for DM. For more information, please visit OctapharmaUSA.com.

About Octagam^® 10%

Octagam^® 10% [Immune Globulin Intravenous (Human)] is an immune globulin intravenous (human) liquid preparation indicated for the treatment of:

• Chronic immune thrombocytopenic purpura (ITP) in adults
• Dermatomyositis (DM) in adults

Warnings

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.