The EMA has recently published their recommendation for extending the indication of a treatment called Nucala to include patients diagnosed with relapsing-remitting or refractory eosinophilic granulomatosis with polyangiitis (EGPA). Specifically, they recommend this therapy as an add-on therapy for individuals who are at least 6 years old.
EGPA is a rare condition that leads to chronic inflammation in the body’s arteries and veins. This inflammation is called vasculitis. The inflammation wreaks havoc on the body affecting the gastrointestinal system, the nervous system, the lungs, heart, and even the skin.
The most common therapy for EGPA is currently corticosteroids. Unfortunately, long-term use of these steroids has detrimental side effects. Side effects can include the thinning of the bones, increased susceptibility to infection, high blood sugar, weight gain, and eye issues. Other therapies include immunosuppressants, which come with their own issues.
Clearly, new treatment options are needed for this patient population.
Nucala
Nucala uses Mepolizumab, a monoclonal antibody, to reduce the number of eosinophils produced in the body. It works by attaching to the IL-5 protein, which is responsible for making the eosinophils. With a reduction of eosinophils, inflammation is reduced. Reduction of inflammation leads to a reduction of symptoms.
The EMA recommending Nucala is thanks to a randomized and placebo-controlled investigation which studied both the safety and the efficacy of the treatment for EGPA. The trial included 136 individuals who were all currently taking corticosteroids.
The trial lasted 52 weeks. Each patient was either given a 300mg dose of Nucala or a 300mg dose of placebo. The treatment was administered subcutaneously. Doses were given every 4 weeks in addition to the corticosteroids. However, after week 4, the corticosteroid dose began to be tapered.
The study found that the group who was given Nucala had a much greater time in remission at week 36 as well as week 48. This means they had a longer period without symptoms or other signs of disease.
The most common side effects that patients experienced were neck pain, infections, respiratory disorders, hypersensitivity, reactions to the administration, and headaches.
Looking Forward
A post-approval study should begin soon as a part of the recommendation. This study will include children from ages 6 to 17.
The next step is for the European Commission to adopt a decision regarding marketing authorization. Following marketing authorization, price decisions must be made.
The goal is, as always, to get patients the treatment as quickly as possible.
You can read more about this recommendation here.
