XOMA Ltd., a lcompany involved in the discovery and development of therapeutic antibodies, announced that the FDA has designated XOMA 052, an antibody to interleukin-1 beta, an orphan drug for the treatment of Behcet’s disease. As previously announced, the Committee for Orphan Medical Products of the European Medicines Agency has recommended the granting of orphan medicinal product designation for XOMA 052 for the same indication in the European Union.
U.S. orphan drug designation is granted by the FDA Office of Orphan Drug Products Development to novel drugs or biologics that may treat a condition affecting fewer than 200,000 persons in the United States.
The designation offers a number of potential incentives, which may include, among others, a seven-year period of U.S. marketing exclusivity from the date of marketing authorization, written guidance on the non-clinical and clinical studies needed to obtain marketing approval, and tax credits for certain clinical research.
In June 2010, XOMA announced positive results from an open-label pilot study of XOMA 052 in Behcet’s disease patients who were suffering from vision-threatening disease exacerbations, known as uveitis, despite maximal doses of immunosuppressive medicines. All seven patients who enrolled in the trial displayed rapid reduction of intraocular inflammation and improvement in visual acuity or other ophthalmic measures following a single treatment with XOMA 052. Five patients received a second infusion to blunt a developing exacerbation, and all responded to the second infusion. The drug appeared to be safe, and no drug-related adverse events were reported. Preliminary results were presented at the Annual Congress of the European League Against Rheumatism (EULAR) in Rome, Italy and at the International Congress on Behcet’s Disease in London in July.