The Food and Drug Administration (FDA) has approved Nucala (mepolizumab; GlaxoSmithKline) for the treatment of adults with eosinophilic granulomatosis with polyangiitis (EGPA), a rare autoimmune disease that causes vasculitis. This approval marks the first FDA-approved treatment for EGPA.
“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 the 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.”
The approval was based on data from a 52-week trial where patients were randomized to receive either Nucala 300mg or placebo subcutaneously once every 4 weeks while continuing oral corticosteroid (OCS) therapy (OCS was tapered starting at week 4). Disease remission while on an OCS dose of ≤4mg of prednisone was considered the primary efficacy outcome.
The 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. Moreover, significantly more patients who received Nucala achieved remission within the first 24 weeks and remained in remission for the remainder of the 52-week study.
Headache, injection site reaction, back pain, and fatigue were the most common adverse reactions associated with Nucala treatment in clinical trials.
Nucala is an interleukin-5 antagonist monoclonal antibody (IgG1 kappa) produced by recombinant DNA technology in Chinese hamster ovary cells. It was previously approved in 2015 as an add-on maintenance treatment in patients with severe asthma, with an eosinophilic phenotype.
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