艾伯维（AbbVie）5月20日宣布，FDA已授予Humira（修美乐，通用名：adalimumab，阿达木单抗）治疗非感染性中、后或泛葡萄膜炎或慢性非感染性前葡萄膜炎的孤儿药地位。

葡萄膜炎是一组罕见严重炎症性眼科疾病，分为前葡萄膜炎、中间葡萄膜、后葡萄膜炎和全葡萄膜炎，按病程可分为急性和慢性。艾伯维正在调查Humira治疗非感染性葡萄膜炎的疗效和安全性，该临床项目已处于III期开发 。目前，Humira尚未获批用于任何形式的葡萄膜炎。

Humira是艾伯维的旗舰产品处方药，是全球首个获批的肿瘤坏死因子(TNF)-α全人源化单克隆抗体药，获批的适应症已达7个之多，包括：中度至重度类风湿关节炎，中度至重度慢性斑块型银屑病，中度至重度克罗恩病；中度至重度溃疡性结肠炎，强直性脊柱炎，银屑病关节炎，中度至重度多关节型幼年特发性关节炎。

该药在2013年的全球销售额高达106亿美元，位列《2013年最畅销药物排行榜》榜首。

英文原文：

AbbVie (ABBV) Receives Orphan Drug Designation for HUMIRA

NORTH CHICAGO, Ill., May 20, 2014 /PRNewswire/ -- AbbVie (NYSE:ABBV) announced today that the U.S. Food and Drug Administration (FDA) has granted HUMIRA® (adalimumab) orphan drug designation for the treatment of non-infectious intermediate, posterior, or pan-uveitis, or chronic non-infectious anterior uveitis, a group of rare but serious inflammatory diseases of the eye. AbbVie is investigating the efficacy and safety of HUMIRA for the treatment of non-infectious uveitis, and the clinical program is in Phase III development. HUMIRA is not currently approved to treat any form of uveitis.

Uveitis is a general term that encompasses several inflammatory eye diseases. The associated inflammation causes damage of eye tissue leading to reduced vision and/or vision loss. While the exact cause of uveitis is unknown, this condition can be caused by an infection, autoimmune disease, medication, surgery or trauma to the eye. Symptoms of uveitis may include vision loss, blurred vision, eye pain and redness, as well as sensitivity to light.[1] It is estimated that uveitis accounts for 10 to 15 percent of all cases of total blindness in the U.S.[2]

"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," said Scott Brun, M.D., vice president, Pharmaceutical Development, AbbVie. "AbbVie remains committed to the ongoing development of HUMIRA to treat a variety of autoimmune diseases where patients have the potential to benefit."

About Orphan Drug Designation 
The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the United States, or that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug.

The approval of an orphan designation request does not alter the standard regulatory requirements and process for obtaining marketing approval for an investigational use. Sponsors must establish safety and efficacy of a compound in the treatment of a disease through adequate and well-controlled studies.

U.S. Product Information for HUMIRA® (adalimumab)

HUMIRA is a prescription medicine used:

To reduce the signs and symptoms of:
   Moderate to severe rheumatoid arthritis (RA) in adults. HUMIRA can be used alone, with methotrexate, or with certain other medicines. HUMIRA may prevent further damage to bones and joints and may help the ability to perform daily activities.
   Moderate to severe polyarticular juvenile idiopathic arthritis (JIA) in children 4 years of age and older. HUMIRA can be used alone, with methotrexate, or with certain other medicines.
Psoriatic arthritis (PsA) in adults. HUMIRA can be used alone or with certain other medicines. HUMIRA may prevent further damage to bones and joints and may help the ability to perform daily activities.
   Ankylosing spondylitis (AS) in adults.
   Moderate to severe Crohn's disease (CD) and to achieve and maintain clinical remission in adults who have not responded well to conventional treatments. HUMIRA is also used to reduce signs and symptoms and achieve clinical remission in these adults who have also lost response to or are unable to tolerate infliximab.
   In adults, to help get moderate to severe ulcerative colitis (UC) under control (induce remission) and keep it under control (sustain remission) when certain other medicines have not worked well enough. It is not known if HUMIRA is effective in people who stopped responding to or could not tolerate anti-TNF medicines.
  To treat moderate to severe chronic plaque psoriasis (Ps) in adults who are ready for systemic therapy or phototherapy, and are under the care of a doctor who will decide if other systemic therapies are less appropriate.