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GSK submits US regulatory filing to expand the use of Nucala in children with severe eosinophilic asthma

GlaxoSmithKline plc (GSK) today announced the filing of a supplemental Biologics License Application (sBLA) to the US Food and Drug Administration (FDA) seeking an additional indication for the use of Nucala (mepolizumab) as an add-on treatment for severe eosinophilic asthma in paediatric patients aged six to 11 years.

Issued: London, UK

The submission is supported by a paediatric open-label study conducted in children aged six to 11 years that investigated pharmacokinetics, pharmacodynamics and long-term safety.

Mepolizumab, a humanised anti-IL5 monoclonal antibody, was approved in the US in 2015 for use as an add-on treatment for patients, aged 12 years or older, with severe asthma and an eosinophilic phenotype. There are currently no targeted biologic therapies available in the US for patients with severe eosinophilic asthma who are as young as six years old.

About severe asthma and eosinophilic inflammation  

Severe asthma is defined as asthma which requires treatment with medium or 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 categorised 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. Interleukin-5 (IL-5) is the main promoter of eosinophil growth, activation and survival and provides an essential signal for the movement of eosinophils from the bone marrow into the lung. Studies suggest that approximately 60% of patients with severe asthma have eosinophilic airway inflammation. While severe eosinophilic asthma is not as prevalent amongst paediatric patients, the burden can be greater when compared with adults and new treatment options are needed to improve asthma control and quality of life.

For more information please see GSK’s infographic about severe asthma and role of eosinophils

About Nucala (mepolizumab)

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.

Mepolizumab has been developed for the treatment of diseases that are driven by inflammation caused by eosinophils. It has been studied in over 3,000 patients in 16 clinical trials across a number of eosinophilic indications. It has been approved (under the brand name Nucala) in the US, Europe and in over 20 other markets as an add-on maintenance treatment for patients with severe eosinophilic asthma and is the leading biologic in this indication. In the EU, in addition to the adult licence it has a licence for paediatric use (from aged six-17 years) in severe eosinophilic asthma. In the US, Japan, Canada and Argentina it is also approved as add-on maintenance treatment for patients with eosinophilic granulomatosis with polyangiitis (EGPA). Mepolizumab is currently being investigated for severe hypereosinophilic syndrome, nasal polyposis and COPD.

About Nucala (mepolizumab) paediatric programme

The paediatric programme was comprised primarily of an open-label, uncontrolled clinical trial conducted in children aged six to 11 years that investigated subcutaneous mepolizumab pharmacokinetics, pharmacodynamics and long-term safety. Additional pharmacokinetics, pharmacodynamics and safety data in children under 18 years old in other indications were also included in the data package. The safety profile in paediatric patients aged six to 11 years was determined to be similar to the safety profile in patients aged 12 years and older.

GSK’s commitment to respiratory disease

GSK has led the way in developing innovative medicines to advance the management of asthma and COPD for nearly 50 years. Over the last five years we have launched six innovative medicines responding to continued unmet patient need, despite existing therapies. This is an industry-leading portfolio in breadth, depth and innovation, developed to reach the right patients, with the right treatment.

Important Safety Information for Nucala

The following information is based on the US Prescribing Information.

Nucala should not be administered to patients with a history of hypersensitivity to mepolizumab or excipients in the formulation.

Hypersensitivity reactions (e.g., anaphylaxis, angioedema, bronchospasm, hypotension, urticaria, rash) have occurred with Nucala. These reactions generally occur within hours of administration but can have a delayed onset (i.e., days). If a hypersensitivity reaction occurs, discontinue Nucala.

Nucala should not be used to treat acute asthma symptoms, acute exacerbations, or acute bronchospasm.

Herpes zoster infections have occurred in patients receiving Nucala. Consider vaccination if medically appropriate.

Do not discontinue systemic or inhaled corticosteroids abruptly upon initiation of therapy with Nucala. Decreases in corticosteroid doses, if appropriate, should be gradual and under the direct supervision of a physician. Reduction in corticosteroid dose may be associated with systemic withdrawal symptoms and/or unmask conditions previously suppressed by systemic corticosteroid therapy.

Treat patients with pre-existing helminth infections before initiating therapy with Nucala. If patients become infected while receiving Nucala and do not respond to anti-helminth treatment, discontinue Nucala until infection resolves.

The most common adverse reactions (≥3% and more common than placebo) reported in the first 24 weeks of 2 asthma clinical trials with 100 mg of Nucala were: headache, injection site reaction, back pain, fatigue, influenza, urinary tract infection, abdominal pain upper, pruritus, eczema, and muscle spasms. In 3 clinical trials, the percentages of subjects who experienced systemic (allergic and nonallergic) reactions were 3% for 100 mg of Nucala and 5% for placebo. Manifestations included rash, flushing, pruritus, headache, and myalgia. A majority of the systemic reactions were experienced on the day of dosing. Injection site reactions (e.g., pain, erythema, swelling, itching, burning sensation) occurred in 8% of subjects treated with 100 mg of Nucala versus 3% treated with placebo.

A pregnancy exposure registry monitors pregnancy outcomes in women with asthma exposed to Nucala during pregnancy. To enrol call 1-877-311-8972 or visit www.mothertobaby.org/asthma. The data on pregnancy exposures from the clinical trials are insufficient to inform on drug-associated risk. Monoclonal antibodies, such as mepolizumab, are transported across the placenta in a linear fashion as the pregnancy progresses; therefore, potential effects on a foetus are likely to be greater during the second and third trimesters.

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Cautionary statement regarding forward-looking statements
GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described under Item 3.D Principal risks and uncertainties in the company's Annual Report on Form 20-F for 2017.