The US Food and Drug Administration (FDA) has expanded the approval of Nucala® (mepolizumab; GlaxoSmithKline) to treat adults with the rare autoimmune disease eosinophilic granulomatosis with polyangiitis (EGPA), which causes vasculitis. This new indication is the first FDA-approved therapy for treating EGPA.
“Prior to today’s action, patients with this challenging, rare disease did not have an FDA-approved treatment option,” Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, said in a press release. “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 FDA granted this application Priority Review and Orphan Drug designations.
Nucala is administered by a healthcare professional as an injection into the upper arm, thigh, or abdomen at a frequency of once every 4 weeks. It was approved in 2015 to treat patients aged 12 years and older for severe asthma with an eosinophilic phenotype, including patients already receiving other asthma medications. The drug is an interleukin-5 antagonist monoclonal antibody (IgG1 kappa) produced by recombinant DNA technology in Chinese hamster ovary cells.
Safety and efficacy of the drug was established in a 52-week clinical trial comparing Nucala to placebo. Patients receiving Nucala 300 mg once every 4 weeks in addition to their stable daily oral corticosteroid therapy achieved significantly greater accrued time in remission compared with those receiving placebo. Findings from the study also showed that significantly more patients receiving Nucala 300 mg achieved remission in the first 24 weeks and remained in remission for the 52 weeks of the study compared with those receiving placebo.
The most common adverse reactions associated with Nucala in clinical trials included headache, injection site reaction, back pain, and fatigue. Hypersensitivity reactions have also been reported, including anaphylaxis, angioedema, bronchospasm, hypotension, urticaria, and rash. Patients should discontinue Nucala if a hypersensitivity reaction occurs. Nucala should not be given to patients with a history of hypersensitivity to mepolizumab or any of its ingredients.
Nucala should not be used to treat acute bronchospasm or status asthmaticus. Patients beginning treatment with Nucala should not discontinue systemic or inhaled corticosteroids abruptly; gradual decrease is advised, if appropriate.
FDA approves first drug for eosinophilic granulomatosis with polyangiitis, a rare disease formerly known as the Churg-Strauss syndrome [press release]. FDA. Published December 12, 2017. Accessed December 12, 2017.