FDA approves first targeted biologic mepolizumab for severe Pediatric Eosinophilic Asthma

The US Food and Drug Administration (FDA) has approved Nucala (mepolizumab), worlds first and only targeted biologic approved for use in 6-11 years old children with severe eosinophilic asthma. Nucala is the only targeted biologic approved by the Food and Drug Administration for treatment of severe eosinophilic asthma.

Severe asthma is defined as asthma which requires treatment with high dose inhaled corticosteroids (ICS) plus a second controller (and/or systemic corticosteroids) to prevent it from becoming 'uncontrolled' or which remains 'uncontrolled' despite this therapy. Severe asthma patients are also often categorized by long-term use of oral corticosteroids (OCS). In a sub-set of severe asthma patients, the over-production of eosinophils (a type of white blood cell) is known to cause inflammation in the lungs.

First approved in 2015 for severe eosinophilic asthma, mepolizumab is the first-in-class monoclonal antibody that targets IL-5. It is believed to work by preventing IL-5 from binding to its receptor on the surface of eosinophils. Inhibiting IL-5 binding in this way reduces blood eosinophils without completely depleting them.

Nucala (100mg dose subcutaneous injection) approved in 2015 is an add-on maintenance treatment for patients with severe eosinophilic asthma aged 12 years and older. This approval (40mg dose subcutaneous injection) extends the current indication in the US for Nucala to patients aged six to 11 years.

Dr Hal Barron, Chief Scientific Officer and President, R&D, GSK, said: "Children with severe eosinophilic asthma currently have limited treatment choices available to them. We believe this important new indication for Nucala is a significant development for these children and their families".

Tonya Winders, CEO, and President, Allergy and Asthma Network, noted: "As a mother of children who suffer from asthma, I know first-hand the huge impact it has on a family, from the constant worry about your child being hospitalized to practical issues like arranging time off work to care for them. Having Nucala approved as the first biologic for treating severe eosinophilic asthma in this young age group represents a significant step forward for the asthma community."

The Food and Drug Administration approval is supported by an open-label study, conducted in children aged six to 11 years and suffering from severe eosinophilic asthma, that investigated Nucala's pharmacokinetics, pharmacodynamics and long-term safety. Evidence from adequate and well-controlled trials in adults and adolescents also supported approval in this age group. The 52-week long-term phase of the study showed that the safety profile in paediatric patients aged six to 11 years was similar to the known safety profile in patients aged 12 years and older.

Dr Daniel Jackson, MD, Department of Pediatrics, University of Wisconsin, added: "Severe eosinophilic asthma in children is a complicated condition that can be extremely challenging to treat. Nucala has made a difference for many adults and adolescents living with severe asthma. This approval is an important development, giving physicians like me a much-needed option to consider for our paediatric patients".

Nucala has been approved for use as an add-on treatment for severe eosinophilic asthma in patients aged six years and older in the EU since August 2018.

In the US, Nucala (100mg subcutaneous injection of mepolizumab) is currently licensed as an add-on maintenance treatment for patients with severe eosinophilic asthma aged 12 years and older. This now extends to patients aged six to 11 years (40mg dose subcutaneous injection). Nucala (300mg as three separate 100mg subcutaneous injections of mepolizumab) is licensed for the treatment of adult patients with eosinophilic granulomatosis with polyangiitis (EGPA). Nucala is not approved for the relief of acute bronchospasm or status asthmaticus.