FDA approves Nucala (mepolizumab) 40 mg prefilled syringe for children with severe eosinophilic asthma

Issued: PHILADELPHIA, PA

GlaxoSmithKline plc (GSK) today announced that the US Food and Drug Administration (FDA) has approved a 40 mg prefilled syringe of Nucala (mepolizumab) for appropriate children aged 6 to 11 years old who have severe eosinophilic asthma (SEA). Nucala can now be given by a child’s health care provider or administered at home by a caregiver once trained by a health care professional. Nucala is an add-on, prescription maintenance treatment for patients 6 years and older with severe eosinophilic asthma.

Previously, children aged 6 to 11 years old received a dose of 40 mg Nucala using a solution that was mixed and administered in a physician’s office. Now, a child’s healthcare provider will determine if at-home administration is appropriate, and if so, will provide instruction to a patient’s caregiver on how to properly administer and monitor for any allergic reactions. Nucala will be administered every four weeks whether at home or in the physician’s office.

Asthma is the most common chronic disease in children.^[i]  It is estimated that 6 million children in the US are living with asthma.^[ii] Approximately 2.5-5% of these cases are characterized as severe.^[iii],[iv] Severe asthma can have an impact on a patient’s quality of life, as their asthma symptoms can remain uncontrolled, despite high-dose standard treatments.^[v],[vi] This can leave patients at an increased risk of asthma attacks.^[vii]

“The younger population with severe eosinophilic asthma often has more symptoms, less control over those symptoms and experiences more frequent exacerbations, making childhood activities challenging,” said Tonya Winders, CEO & President, Allergy and Asthma Network and President of Global Allergy and Airways Patient Platform. In addition, she emphasized “for many, going to the doctor’s office to receive a biologic can be challenging, so having the possibility for children to receive Nucala at home, provides a bit more flexibility for the child’s and caregiver’s lives.”

“The needs of our patients and physicians continue to be the driving force of our innovation and new treatment offerings in the respiratory space,” said Dr. Tom Corbridge, GSK U.S. Senior Medical Lead. “Being able to provide a treatment option administered at home for patients as young as 6 years old who are living with severe eosinophilic asthma, is an example of how our commitment to the community combined with clinical excellence can deliver on those needs.”

Through ongoing research, GSK is committed to improving the lives of those living with disease associated with uncontrolled eosinophilic inflammation, continuously innovating to address the unmet needs in this patient group.

About Nucala (mepolizumab)

Nucala (mepolizumab) is indicated for the add-on maintenance treatment of adult and pediatric patients aged 6 years and older with severe asthma and with an eosinophilic phenotype. Nucala is not indicated for the relief of acute bronchospasm or status asthmaticus. First approved in 2015 for SEA, Nucala was 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, reducing blood eosinophils and maintaining them within normal levels. The mechanism of action for mepolizumab has not been definitively established.

Nucala is available as a solution in a prefilled syringe, prefilled autoinjector and as a lyophilized powder that comes in a vial and is reconstituted for injection for in-office use. If their healthcare professional determines that it is appropriate, people with SEA (adults and pediatrics aged 6 years and older) and their caregivers can administer Nucala at home, after training by a health professional.

Nucala has been developed for the treatment of diseases that are driven by inflammation associated with eosinophils. It has been studied in over 4,000 patients in 41 clinical trials across several eosinophilic indications and has been approved in the US, the EU and in over 25 other markets, as an add-on maintenance treatment for patients with SEA. Mepolizumab is approved in 17 markets, including the EU and US, for pediatric use in SEA from ages six to 17 years of age, with approval in an additional seven markets for use in patients with SEA aged 12-17 years. The first approval for mepolizumab in chronic rhinosinusitis with nasal polyps (CRSwNP) was granted by the FDA in July 2021. Mepolizumab is approved for use in patients with eosinophilic granulomatosis with polyangiitis (EGPA) in a total of 14 markets including the US, Japan and Canada.  Mepolizumab was first approved for use in hypereosinophilic syndrome (HES) in the US in September 2020 and approvals have since then been granted in an additional 5 markets.

In addition, mepolizumab is being studied in patients with chronic obstructive pulmonary disease (COPD) with an eosinophilic phenotype and it is not currently approved for use in COPD anywhere in the world.

About severe asthma and 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 can also be defined by the need for maintenance use of oral corticosteroids (OCS).^[viii]

SEA is characterized by having an increase of eosinophils, a type of white blood cell that helps fight disease and infections. However, in some people, a high number of eosinophils can have a negative effect and is associated with inflammation of the airways, potentially resulting in asthma symptoms.⁶

Nucala is indicated in the US for the:

• add-on maintenance treatment of adult and pediatric patients aged 6 and older with severe asthma and with an eosinophilic phenotype. NUCALA is not used to treat sudden breathing problems.
• add-on maintenance treatment of CRSwNP in adults whose disease is not controlled with nasal corticosteroids.
• treatment of adult patients with EGPA.
• treatment of adult and pediatric patients aged 12 years and older with HES for ≥6 months without an identifiable non-hematologic secondary cause.

Important safety information

The following information is based on the US Prescribing Information for Nucala in licensed indications only. Please consult the full Prescribing Information for all the labelled safety information for Nucala.

CONTRAINDICATIONS

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

WARNINGS AND PRECAUTIONS

• 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.

ADVERSE REACTIONS

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 100 mg 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. 

In a clinical trial in patients with hypereosinophilic syndrome, no additional adverse reactions were identified to those reported in the severe asthma trials. Injection site reactions (e.g., burning, itching) occurred in 7% of subjects treated with 300 mg of Nucala versus 4% treated with placebo.

In a clinical trial with CRSwNP, the most common adverse reactions (incidence ≥/= 5%) in patients receiving NUCALA 100 mg was oropharyngeal pain and arthralgia. Injection site reactions (e.g., erythema, pruritus) occurred in 2% of patients receiving NUCALA versus <1% treated with placebo.

GSK’s commitment to respiratory disease

For over 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.  

About GSK

GSK is a science-led global healthcare company. For further information please visit www.gsk.com/about-us.

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 in the Company's Annual Report on Form 20-F for 2020 and any impacts of the COVID-19 pandemic.

[i] American Lung Association. Asthma and Children Fact Sheet. Retrieved from: https://www.lung.org/lung-health-diseases/lung-disease-lookup/asthma/learn-about-asthma/asthma-children-facts-sheet. Last Accessed January 2022.

[ii] Centers for Disease Control and Prevention. Asthma in Children. U.S. Department of Health & Human Services. www.cdc.gov/vitalsigns/childhood-asthma/index.html. Last Accessed January 2022.

[iii] Brusselle G, Koppelman G. Biologic Therapies for Severe Asthma. N Engl J Med 2022; 386:157-171. Published 2022 Jan 13. DOI: 10.1056/NEJMra2032506

[iv] Guilbert TW, Bacharier LB, Fitzpatrick AM. Severe asthma in children. J Allergy Clin Immunol Pract. 2014;2(5):489-500. doi:10.1016/j.jaip.2014.06.022.

[v] Hossny E, Caraballo L, Casale T, El-Gamal Y, Rosenwasser L. Severe asthma and quality of life. World Allergy Organ J. 2017;10(1):28. Published 2017 Aug 21. doi:10.1186/s40413-017-0159-y

[vi] American Lung Association. Severe Asthma. Retrieved from: https://www.lung.org/lung-health-diseases/lung-disease-lookup/asthma/learn-about-asthma/severe-asthma. Last Accessed January 2022.

[vii] National Library of Medicine. Asthma. Retrieved from: https://medlineplus.gov/asthma.html.

[viii] Chung et al. International ERS/ATS guidelines on definition, evaluation and treatment of severe asthma. European Respiratory Journal Feb 2014, 43(2) 343-373; DOI: 1183/09031936.00202013.