GlaxoSmithKline has announced today that both all endpoints were met in a pivotal phase III study of mepolizumab for rare disease Eosinophilic Granulomatosis with Polyangiitis (EGPA), approved as Nucala as an add-on treatment for some types of asthma in adult patients, and teenage patients in the U.S.
This study inspected IL-5 antagonist in patients with relapsing and refractory EGPA, a rare disease characterised by widespread inflammation in the walls of small blood vessels (vasculitis).
A key goal of treatment for EGPA is to induce and maintain remission while reducing the use of corticosteroids and other immunosuppressive therapies.
Mepolizumab showed better than placebo in the study for all secondary endpoints.
Steve Yancey, Vice President and Medicine Development Lead for mepolizumab, GSK said: “We are very pleased to observe the positive benefits of treatment with mepolizumab across several clinically relevant measures in this first ever double-blind, placebo-controlled study in patients with Eosinophilic Granulomatosis with Polyangiitis. Given that patients with this rare systemic inflammatory disease have limited treatment options, these results represent a significant step forward in our efforts to help them. We now look forward to progressing our regulatory submission plans.”
One death in the study
Most frequent on-treatment serious adverse events reported for mepolizumab and placebo, respectively were asthma (4%, 4%), influenza (0, 3%) and pneumonia (0, 3%). One death was reported in a patient receiving mepolizumab which was not considered by the investigator to be related to study treatment.
Where is this mepolizman approved and for what?
Mepolizumab is not approved for use anywhere in the world for EGPA, except for use in the E.U., under the brand name Nucala, for use as an add-on treatment for severe refractory eosinophilic asthma in adult patients.
Nucala is approved for use in the U.S. as an add-on maintenance treatment of patients with severe asthma aged 12 years and older, and with an eosinophilic phenotype. It is not approved for the treatment of other eosinophilic conditions or relief of acute bronchospasm or status asthmaticus.
Nucala has also been approved in Canada, Australia, Japan, Switzerland, Chile, South Korea and Taiwan.