Seeking approval: present and future therapies for pemphigus vulgaris

Abstract

Pemphigus vulgaris is an autoimmune blistering disease of the skin and mucous membranes. Despite the potentially fatal prognosis, there are currently no FDA-approved treatments specifically for pemphigus. In 2006, the FDA designated orphan drug status to mycophenolate mofetil for the treatment of pemphigus vulgaris, indicating both federal and commercial interest in developing therapies for this devastating disease. This review focuses on pemphigus therapies that are currently in preclinical or clinical trials, as well as potential novel therapies based on recent advances in the understanding of the pathophysiology of this disease.