Autoimmune blistering diseases: promising agents in clinical trials

Abstract

Introduction: Treatment options for autoimmune bullous diseases (AIBD) are currently limited to corticosteroids and traditional immunomodulants and immunosuppressants that are associated with unfavorable adverse effect profiles. The most frequent AIBDs, i.e. bullous pemphigoid, pemphigus vulgaris, and mucous membrane pemphigoid, impose a high disease burden onto affected patients and can be detrimental due to infections, exsiccosis, and impaired food intake. Significant progress has been made in elucidating disease mechanisms and key mediators by in vivo and in vitro models, thus identifying a multifaceted range of possible drug targets. However, except for rituximab for pemphigus vulgaris, no new drugs have been approved for the treatment of AIBDs in the last decades.

Areas covered: This review covers new drug developments and includes ongoing or completed phase 2 and 3 clinical trials. Studies were identified by querying the registries of ClinicalTrials.gov and Cochrane Library.

Expert opinion: Promising results were shown for a variety of new agents including nomacopan, efgartigimod, omalizumab, dupilumab, as well as chimeric autoantibody receptor T cells. Clinical translation in the field of AIBDs is highly active, and we anticipate significant advances in the treatment landscape.

Keywords: autoimmune bullous diseases; bullous pemphigoid; chimeric autoantigen receptor; dupilumab; efgartigimod; mucous membrane pemphigoid; nomacopan; pemphigus vulgaris.