Rare Disease Report

FDA Expands Approval of Nucala to Include Treatment of Churg-Strauss Syndrome

DECEMBER 12, 2017
Mathew Shanley
This morning, the U.S. Food and Drug Administration (FDA) expanded the approved use of Nucala (mepolizumab) to treat adult patients with eosinophilic granulomatosis with polyangiitis (EGPA).

A study published in the New England Journal of Medicine last May found that treatment with the asthma medication provided clinical benefit to approximately half of patients with refractory or relapsed EGPA – a patient population that has been historically difficult to treat.

EGPA, commonly referred to as Churg-Strauss syndrome, is a rare autoimmune disease characterized by asthma, high levels of eosinophils, an inflammation of small- to medium-sized blood vessels, and its ability to cause vasculitis. The inflamed vessel can cause life-threatening damage to the lungs, gastrointestinal tract, skin, heart, and nervous system, and the condition affects an estimated 10-14 people per every 1-million.

Mepolizumab is a humanized IgG monoclonal antibody specific to interleukin 5 (IL-5) that binds to the gene and prevents it from attaching to its receptor on the surface of eosinophils. By inhibiting IL-5, the drug reduces blood, tissue and sputum eosinophil levels, which can reduce severe inflammation.

In November 2016, a Phase 3 trial evaluating GlaxoSmithKline’s drug met its primary endpoints, as it exhibited total duration of remission. The 52-week study assed a 300mg dosage of the drug in 136 patients with relapsing or refractory EGPA who were receiving standard-of-care therapy. Until today’s approval, standard-of-care for this patient population has been systemic glucocorticoids. In patients who cannot receive this therapy, however, or in patients who relapse, immunosuppressive agents have been used.

Patients who received this dose of mepolizumab achieved a significantly greater accrued time in remission in comparison to patients who received placebo.

“Prior to today’s action, patients with this challenging, rare disease did not have an FDA-approved treatment option,” said Badrul Chowdhury, M.D., Ph.D., director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research in the official FDA 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 original approval of Nucala was in 2015 and for the treatment of patients age 12 and older with a specific subgroup of asthma. The new indication recommends administration once every 4 weeks via subcutaneous injection by a health care professional into the patient’s upper arm, thigh, or abdomen.

Adverse events (AEs) reported in patients who have received Nucala in clinical trials include: headache, injection site reaction, back pain, and fatigue.

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