Mepolizumab gets the nod for the rare disease formerly known as Churg-Strauss syndrome.
Mepolizumab (Nucala) is the first drug approved by the FDA specifically to treat adult patients with eosinophilic granulomatosis with polyangiitis (EGPA).
According to the National Institutes of Health, EGPA affects 10.7 to 14 per 1,000,000 adults. This rare autoimmune disease was formerly known as the Churg-Strauss syndrome.
Mepolizumab is an interleukin-5 antagonist monoclonal antibody (IgG1 kappa) produced by recombinant DNA technology in Chinese hamster ovary cells. It is administered once every 4 weeks by subcutaneous injection by a health care professional. The medication was previously approved by the FDA in 2015 to treat patients older than 12 with a specific subset of asthma (severe asthma with an eosinophilic phenotype) despite treatment with their current asthma medications.
The expanded indication of mepolizumab meets a critical, previously unmet need for patients with EGPA. “Prior to today’s action, patients with this challenging, rare disease did not have an FDA-approved treatment option,” said Badrul Chowdhury, Director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research, in a recent FDA press announcement.
The safety and efficacy of mepolizumab was based on data from a 52-week treatment clinical trial that compared mepolizumab with placebo. Patients receiving mepolizumab achieved a significantly greater accrued time in remission compared with those receiving placebo. Significantly more patients who received mepolizumab also achieved remission within the first 24 weeks and remained in remission for the remainder of the 52-week study period, as compared with patients who received the placebo.
The most common adverse reactions associated with mepolizumab included headache, injection site reaction, back pain, and fatigue.
US Food & Drug Administration. FDA approves first drug for eosinophilic granulomatosis with polyangiitis, a rare disease formerly known as the Churg-Strauss syndrome. Published online December 12, 2017.