FDA Grants Orphan Drug Designation to Octapharma USA’s Experimental Dermatomyositis Drug Octagam


The investigational drug intravenous immunoglobulin, or IVIG (brand name Octagam), under development by Octapharma USA to treat dermatomyositis, has received U.S. Food and Drug Administration (FDA) orphan drug designation.

IVIG therapy involves the injection of a pool of nonspecific antibodies (immunoglobulin) that may work by dialing down the immune system’s production of its own antibodies, much as warm air tells a thermostat to stop pumping out heat. It is used in the treatment of a wide variety of autoimmune or inflammatory conditions.

What this means for individuals and families

Orphan drug designation may help speed development of Octagam for the treatment of dermatomyositis, as it provides incentives meant to encourage Octapharma to develop and market it.

Octapharma currently is conducting a phase 3 clinical trial to test the safety and efficacy of Octagam in dermatomyositis. Trial sites are located the U.S. in Arizona, Kansas, Michigan, New York, Ohio and Pennsylvania. For more information, visit ClinicalTrials.gov and enter NCT02728752 in the search box.

What this means for Octapharma

Orphan Drug Designation is important to companies, like Octapharma, that are developing therapies because it makes them eligible for special incentives that have been put into place to encourage the development of treatments for rare disorders. In the U.S., a disorder is considered “rare” if it affects fewer than 200,000 individuals — and all the diseases under MDA’s umbrella are considered rare under this definition.

When a potential therapy receives this designation, the sponsor (i.e. the company who is seeking approval from the FDA) can benefit from incentives including reduced taxes, reduced or waived fees for filing with the FDA, and additional years of market exclusivity (when they are protected by law from competition), among others.

The purpose of the orphan drug designation incentives is to encourage companies to work in the rare disease space where the high costs associated with developing a therapy are less likely to be recouped if the treatment is approved and marketed due to the small patient population that may be eligible to benefit from the therapy.

Because dermatomyositis impacts fewer than 200,000 individuals in the U.S. and because the other orphan drug designation requirements were satisfied by Octapharma, this therapy will have the protections and benefits of orphan drug status.