First Treatment Approved by FDA to Treat EGPA

Nucala (mepolizumab, GlaxoSmithKline) has been approved by the U.S. Food and Drug Administration (FDA) to treat eosinophilic granulomatosis with polyangiitis (EGPA), a rare autoimmune disease that causes vasculitis. Approval was based on data from a 52-week trial in which patients were randomized to receive either Nucala 300 mg or placebo subcutaneously once every four weeks while continuing oral corticosteroid (OCS) therapy (OCS was tapered at week four). Results showed that compared with placebo, Nucala-treated patients achieved a significantly greater accrued time in remission, with a higher proportion of patients in the Nucala group achieving remission at both week 36 and week 48. In addition, significantly more patients who received Nucala achieved remission within the first 24 weeks and remained in remission for the 52-week study.

“Prior to today’s action, patients with this challenging, rare disease did not have an FDA-approved treatment option,” said Badrul Chowdhury, MD, PhD, director of the division of pulmonary, allergy and rheumatology products in FDA’s Center for Drug Evaluation and Research. “The expanded indication of Nucala meets a critical, unmet need for EGPA patients. It’s notable that patients taking Nucala in clinical trials reported a significant improvement in their symptoms.”