XOMA's Gevokizumab Wins FDA Orphan Drug Designation
XOMA Corporation announced that the U.S. Food and Drug Administration has granted orphan drug designation to its IL-1 beta modulating antibody labeled gevokizumab for the treatment of pyoderma gangrenosum. XOMA specializes in the discovery and development of therapeutic antibodies such as gevokizumab.
The FDA Office of Orphan Products Development (OOPD) only grants orphan drug designation to novel treatments or biologics which has potential to treat rare diseases or conditions affecting less than 200,000 patients in the U.S. An orphan drug designation gives drugmakers a seven year period of marketing exclusivity in the U.S., privilege to apply for annual grant funding, and clinical research trial design assistance. In addition, a waiver of Prescription Drug User Fee Act (PDUFA) filing fees is granted along with tax credits for clinical research costs.
John Varian, Chief Executive Officer of XOMA, said “Selecting pyoderma gangrenosum as our next Phase 3 indication reflects our commitment to creating and capturing value from gevokizumab, particularly in indications where patients have few effective treatment options… We intend to present what we believe are compelling data from our pilot study in PG and to solicit feedback from the FDA about the requirements for a Phase 3 program in this rare disease.”
Pyoderma gangrenosum is a rare neutrophilic dermatosis of painful spreading necrotic skin ulcers, which can be classified under four types based on skin ulcers manifested. The disease occurs in about 1 per 100,000 people. Up to 50 to 70 percent of PG patient population are estimated to have an underlying systemic condition, with the rest of patients idiopathic. Though combination treatments consisting of topical and systemic therapies are available, up to 46 percent of PG patients experience a relapse of the disease.
Gevokizumab is a monoclonal antibody with unique allosteric modulating properties. The drug has potential to treat patients with various inflammatory diseases such as pyoderma gangrenosum. The drug works by binding to interleukin-1 beta (IL-1 beta) which is a pro-inflammatory cytokine and modulating cellular signaling events that trigger inflammation. Gevokizumab is currently being studied in the global Phase III clinical program EYEGUARD. The company also has an ongoing proof-of-concept program to study gevokizumab’s safety and efficacy in multiple indications.