FDA Grants Orphan Drug Status To AbbVie's HUMIRA For Rare Eye Disease

AbbVie announced that it has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for Humira (adalimumab) as an investigational treatment of certain forms of non-infectious uveitis, a group of rare but serious inflammatory eye diseases.

Uveitis is characterized by associated inflammation that damages eye tissue and leads to reduced vision and/or vision loss. The exact cause of the rare eye disease is not known but can be attributed to infection, medication, autoimmune disease, surgery, or trauma to the eye. The eye disease is responsible for 10 to 15 percent of all cases of total blindness in the U.S.

Scott Brun, VP of Pharmaceutical Development at AbbVie, said, “Few well characterized treatment options are available for patients suffering from uveitis, and the orphan drug designation recognizes the significant unmet need that exists within this disease. AbbVie remains committed to the ongoing development of Humira to treat a variety of autoimmune diseases where patients have the potential to benefit.”

Specifically, the drug received orphan status for the treatment of non-infectious intermediate, posterior, or pan-uveitis, or chronic non-infectious anterior uveitis.

Humira is approved as a prescription medicine used to reduce signs and symptoms of moderate to severe rheumatoid arthritis, moderate to severe polyarticular juvenile idiopathic arthritis (JIA) in children, psoriatic arthritis (PsA), ankylosing spondylitis (AS), moderate to severe Crohn's disease (CD), management of ulcerative colitis, and treatment of moderate to severe chronic plaque psoriasis (Ps). Humira is currently undergoing Phase III clinical development in non-infectious uveitis and is not yet approved to treat any form of uveitis.

Orphan drug designation by the FDA is given to biologics and drugs that are intended to treat, diagnose, or prevent rare diseases/disorders affecting less than 200,000 people in the U.S. Approval of an orphan drug designation does not exert any influence or consequence on the standard regulatory requirements for FDA marketing approval.