XOMA Antibody Receives Orphan Drug Designation from the FDA and EMA

The US Food and Drug Administration has designated XOMA 052, an antibody to interleukin-1 beta manufactured by XOMA, Ltd. (Berkley, CA), an orphan drug for the treatment of Behcet's disease. The Committee for Orphan Medical Products of the European Medicines Agency also has recommended the granting of orphan medicinal product designation for XOMA 052 for the same indication in the European Union (EU).

XOMA 052 is a monoclonal antibody with the potential to improve the treatment of patients with a wide variety of inflammatory diseases. XOMA 052 binds to interleukin-1 beta (IL-1 beta), a pro-inflammatory cytokine involved in diseases including Type 2 diabetes, cardiovascular disease, rheumatoid arthritis, gout, and auto-inflammatory diseases. By binding to IL-1 beta, XOMA 052 inhibits the activation of the IL-1 receptor, thereby preventing the cellular signaling events that produce inflammation.

US 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 US. The designation offers a number of potential incentives, which may include, among others, a seven-year period of US 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.

The EMA's orphan drug program is designed to promote the development of drugs to treat life-threatening or chronically debilitating conditions with a prevalence of up to five in 10,000 persons in the EU. The designation provides EU market exclusivity for up to 10 years following approval for the given indication. Other potential benefits include protocol assistance, direct access to centralized marketing authorization procedures, and financial incentives.