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FDA Expands Approval of Nucala for Eosinophilic Granulomatosis With Polyangiitis
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The FDA has expanded the approved use of Nucala to treat adult patients with eosinophilic granulomatosis with polyangiitis, a rare autoimmune disease that causes vasculitis.
According to an FDA press release, the new indication for Nucala (mepolizumab, GlaxoSmithKline) makes it the first approved therapy specifically to treat eosinophilic granulomatosis with polyangiitis (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 at the FDA Center for Drug Evaluation and Research, said in the release. “The expanded indication of mepolizumab meets a critical, unmet need for EGPA patients. It’s notable that patients taking mepolizumab in clinical trials reported a significant improvement in their symptoms.”
Formally known as Churg-Strauss syndrome, EGPA is characterized by asthma, high levels of eosinophils and inflammation of small- to medium-sized blood vessels. The inflamed vessels can affect various organ systems, including the lungs, gastrointestinal tract, skin, heart and nervous system.
According to the release, the FDA has granted mepolizumab — an interleukin-5 antagonist monoclonal antibody (IgG1 kappa) produced by recombinant DNA technology in Chinese hamster ovary cells — priority review and orphan drug designations, which provide incentives to assist and encourage the development of drugs for rare diseases.
The FDA previously approved mepolizumab for the treatment of patients aged 12 years and older with severe asthma with an eosinophilic phenotype who were already receiving asthma medications. It is administered once every 4 days by subcutaneous injection by a health care professional into the upper arm, thigh or abdomen.
The FDA based its expanded approval on data from a 52-week clinical trial that compared mepolizumab with placebo. Patients received 300 mg of mepolizumab or placebo once every 4 weeks while continuing their stable daily oral corticosteroids therapy. Oral corticosteroids were tapered during the treatment period at week 4. The primary efficacy assessment in the trial measured mepolizumab’s treatment impact on disease remission while on an oral corticosteroid dose of 4 mg or less of prednisone.
According to the FDA, patients receiving mepolizumab achieved a significantly greater accrued time in remission compared with those who received a placebo. A significantly higher proportion of patients receiving mepolizumab achieved remission at both week 36 and week 48 compared with placebo. In addition, significantly more patients who received mepolizumab achieved remission within the first 24 weeks, and remained in remission for the remainder of the 52-week study treatment period compared with patients who received the placebo.
The most common adverse reactions associated with mepolizumab in clinical trials included headache, injection site reaction, back pain and fatigue, the release stated; hypersensitivity reactions, including anaphylaxis, angioedema, bronchospasm, hypotension, urticaria and rash have also occurred. The FDA noted that mepolizumab should not be given to patients with a history of hypersensitivity to mepolizumab or one of its ingredients, and should not be used to treat acute bronchospasm or status asthmaticus. For more information, visit the FDA website here. – by Jason Laday
Disclosure: Chowdhury reports no relevant financial disclosures.