GlaxoSmithKline (LSE/NYSE: GSK) today announced that the US Food and Drug Administration (FDA) has approved Nucala (mepolizumab) for use in children as young as six years old who are living with severe eosinophilic asthma. Nucala is the only targeted biologic to be approved for the condition in the six to 11-year age group in the US.
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 hospitalised, 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 FDA 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 (100mg dose subcutaneous injection) was first approved in 2015 as 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.
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.
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 without completely depleting them.
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 21 clinical trials across a number of eosinophilic indications and is the only biologic with 4.8 years of safety and efficacy data in severe eosinophilic asthma (SEA). Mepolizumab 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. It is also the only anti-IL5 biologic therapy approved for paediatric use from ages six to 17 in Europe in severe eosinophilic asthma. In the US, Japan and Canada and a number of other markets, it is approved as add-on maintenance treatment for patients with EGPA. Mepolizumab is currently being investigated for severe hypereosinophilic syndrome, nasal polyposis and COPD.
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. Full US Prescribing Information is available at US Prescribing Information Nucala.
About severe asthma and eosinophilic inflammation
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 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.
GSK’s commitment to respiratory disease
For 50 years, GSK has led the way in developing medicines that advance the management of asthma and COPD. From introducing the world’s first selective short-acting beta agonist in 1969, to launching six treatments in five years to create today’s industry-leading respiratory portfolio, we continue to innovate so we can reach the right patients, with the right treatment. Working together with the healthcare community, we apply world-class science to discover and understand the molecules that become the medicines of tomorrow. We won’t stand still until the simple act of breathing is made easier for everyone.
Serving paediatric patients
We are committed to bringing our specialty biologic medicines to paediatric patients who have limited treatment options. We are currently progressing regulatory reviews of treatments for children with severe eosinophilic asthma and lupus, reinforcing our commitment to serving an unmet need with these medicines that are proven to be effective in adults.
Important safety information for Nucala (mepolizumab)
The following is a summary of the safety information for Nucala. Please consult the full Prescribing Information, including Patient Information, at www.Nucala.com for all the safety information for Nucala.
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 after administration of Nucala. Discontinue Nucala in the event of a hypersensitivity reaction.
Do not use to treat acute bronchospasm or status asthmaticus.
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. Decrease corticosteroids gradually, if appropriate.
Treat patients with pre-existing helminth infections before therapy with Nucala. If patients become infected while receiving treatment with Nucala and do not respond to anti-helminth treatment, discontinue Nucala until parasitic infection resolves.
Most common adverse reactions (incidence ≥5%) in severe asthma clinical trials included headache, injection site reaction, back pain, and fatigue. Injection site reactions (e.g. pain, erythema, swelling, itching, burning sensation) occurred in 8% of subjects treated with 100mg of Nucala versus 3% treated with placebo.
In a clinical trial in patients with EGPA receiving 300 mg of Nucala, no additional adverse reactions were identified to those reported in severe asthma clinical trials. Injection site reactions (e.g. pain, erythema, swelling) occurred in 15% of subjects treated with 300 mg of Nucala versus 13% treated with placebo.
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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 2018.
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