GSK’s Nucala Approved by FDA as Add-on Therapy for EGPA Patients

The U.S. Food and Drug Administration (FDA) has approved Nucala (mepolizumab) as an add-on to standard care for treating eosinophilic granulomatosis with polyangiitis (EGPA), previously called Churg-Strauss syndrome.

“Patients suffering from EGPA too often face a frustrating journey from a delay in receiving a proper diagnosis to having few effective treatment options with an acceptable safety profile,” Dr. Peter A. Merkel, chief of the Division of Rheumatology at Perelman School of Medicine, University of Pennsylvania & MIRRA study site investigator, said in a press release.

“Rheumatologists, immunologists, and pulmonologists have an important role in properly diagnosing and treating patients with EGPA. Today’s approval of mepolizumab [Nucala] provides specialists with the ability to offer a targeted treatment to appropriate patients with this complex disease.”

The approval is based on results from the Phase 3 MIRRA study (NCT02020889), which included 136 patients with relapsing EGPA or EGPA that did not respond to treatment.

Participants were randomized to receive injections of either Nucala plus standard care – corticosteroids with or without immunosuppressants – or a placebo plus standard care every four weeks for one year.

Length of time in remission – one of the study’s primary endpoints – was significantly longer in the group that received Nucala. Also, the number of patients who achieved remission at both 36 and 48 weeks into the trial was significantly higher in the Nucala group.

The safety of both treatments was similar. Adverse events occurred in 97% of participants in the Nucala group versus 94% in the placebo group. Serious adverse events occurred in 18% of the patients who received Nucala compared with 26% in the placebo group. The most frequent serious adverse event was worsening of asthma.

“Patients with EGPA often suffer from recurrent relapses that place them at greater risk of permanent tissue and organ damage,” said Dr. Michael E. Wechsler, professor of Medicine at National Jewish Health in Denver, Colorado, and principal investigator of the MIRRA study.

“Clinical data demonstrated that mepolizumab increased accrued time in remission, reduced the frequency of relapse and flares, and enabled patients to have their dose of corticosteroid reduced compared to placebo in patients already receiving standard of care. These are key treatment goals and this approval is an important milestone both for treating physicians and for patients,” Wechsler said.

Eric Dube, senior vice president and head, GSK Global Respiratory Franchise, added that “following physician and patient experience with Nucala in severe eosinophilic asthma, we are thrilled that the FDA has expanded the use of this medicine to patients with EGPA, another eosinophil-driven disease, enabling GSK to make it available to patients.”

“This approval follows the positive results of the largest prospective treatment study conducted in EGPA to date, and now for the first time physicians have a targeted treatment option for this debilitating condition,” he said.

Symptoms of EGPA include extreme fatigue, weight loss, muscle and joint pain, sinus and nasal symptoms, and shortness of breath. The disease can cause damage to lungs, skin, heart, sinuses, the gastrointestinal tract, nerves, and other organs.

GSK and the National Institute of Allergy and Infectious Diseases collaborated on the MIRRA study. Patient assistance programs are available from GSK for those who qualify.