Tezepelumab granted Orphan Drug Designation in the US for eosinophilic esophagitis

Tezepelumab has been granted Orphan Drug Designation (ODD) in the US by the Food and Drug Administration (FDA) for the treatment of eosinophilic esophagitis (EoE).

Tezepelumab is being developed by AstraZeneca in collaboration with Amgen and is under Priority Review for patients with asthma in the US. The FDA grants ODD status to medicines and potential new medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US. [1]

Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Eosinophilic esophagitis is a rare disease which involves a range of inflammatory cells that contribute to debilitating symptoms for patients, including severe pain and difficulty swallowing food. There are currently no approved treatments for eosinophilic esophagitis in the US. We are hopeful that tezepelumab, with its unique mechanism of action that targets the top of the inflammatory cascade, could become a potential new medicine to improve outcomes for these patients.”

A decision on tezepelumab’s Priority Review in patients with asthma in the US is expected in the first quarter of 2022. Tezepelumab is also under regulatory review for asthma in the EU and Japan.

EoE is a rare, chronic, inflammatory disease that occurs when eosinophils, a type of white blood cell, accumulate in the esophagus.2 In addition to eosinophils, other cells including mast cells, T-cells and fibroblasts drive injury, inflammation and detrimental tissue remodelling.3 If the disease is not effectively treated it can make eating difficult or uncomfortable, potentially leading to chronic pain, difficulty swallowing, poor growth, malnutrition and weight loss. [2]

The most common symptoms of EoE include reflux that does not respond to medication, difficulty swallowing, food becoming stuck in the esophagus, nausea and vomiting, abdominal or chest pain, poor appetite and difficulty sleeping. [2]

Patients are often treated with corticosteroids to manage inflammation. Currently there are no FDA-approved treatments for EoE. [2]

Tezepelumab is being developed by AstraZeneca in collaboration with Amgen as a potential first-in-class human monoclonal antibody that inhibits the action of thymic stromal lymphopoietin (TSLP), a key epithelial cytokine that sits at the top of multiple inflammatory cascades. Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, which are implicated in the epithelial disease pathophysiology of a number of diseases. [4,5,6,7] Tezepelumab is in development for potential indications including asthma, chronic obstructive pulmonary disease, nasal polyps and chronic spontaneous urticaria. In EoE, a Phase III trial is planned.

Amgen collaboration
In 2020, Amgen and AstraZeneca updated a 2012 collaboration agreement for tezepelumab. Both companies will continue to share costs and profits equally after payment by AstraZeneca of a mid single-digit inventor royalty to Amgen. AstraZeneca continues to lead development and Amgen continues to lead manufacturing. All aspects of the collaboration are under the oversight of joint governing bodies. Under the amended agreement in North America, Amgen and AstraZeneca will jointly commercialise tezepelumab; Amgen will record sales in the US and AstraZeneca will record sales in Canada. AstraZeneca’s share of gross profits from tezepelumab in the US will be recognised as collaboration revenue. In all countries outside the US and Canada, AstraZeneca will solely commercialise tezepelumab. AstraZeneca will record all sales outside of the US as product sales and recognise Amgen’s share of gross profit as cost of sales.

AstraZeneca in Respiratory & Immunology
Respiratory & Immunology, part of BioPharmaceuticals, is one of AstraZeneca’s main disease areas and is a key growth driver for the Company.

AstraZeneca is an established leader in respiratory care with a 50-year heritage. The Company aims to transform the treatment of asthma and COPD by focusing on earlier biology-led treatment, eliminating preventable asthma attacks, and removing COPD as a top-three leading cause of death. The Company’s early respiratory research is focused on emerging science involving immune mechanisms, lung damage and abnormal cell-repair processes in disease and neuronal dysfunction.

With common pathways and underlying disease drivers across respiratory and immunology, AstraZeneca is following the science from chronic lung diseases to immunology-driven disease areas. The Company’s growing presence in immunology is focused on five mid- to late-stage franchises with multi-disease potential, in areas including rheumatology (including systemic lupus erythematosus), dermatology, gastroenterology, and systemic eosinophilic-driven diseases. AstraZeneca’s ambition in Respiratory & Immunology is to achieve disease modification and durable remission for millions of patients worldwide.

AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.

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[1]     US Food and Drug Administration website. Developing Products for Rare Diseases & Conditions. Available at: https://www.fda.gov/industry/developing-products-rare-diseases-conditions. [Last accessed: October 2021].
[2]    American Partnership for Eosinophilic Disorders. EoE. Available at: https://apfed.org/about-ead/egids/eoe. [Last accessed: October 2021].
[3]     O-Shea KM, et al. Pathophysiology of eosinophilic esophagitis. Gastroenterology 2018;154:333-45.
[4]     Menzies-Gow A, et al. Tezepelumab in Adults and Adolescents with Severe, Uncontrolled Asthma. N Engl J Med. 2021; 384: 1800-1809. DOI: 10.1056/NEJMoa2034975.
[5]    Clinicaltrials.gov. Study to Evaluate Tezepelumab in Adults with Chronic Spontaneous Urticaria (INCEPTION). Available at: https://clinicaltrials.gov/ct2/show/NCT04833855. [Last accessed: October 2021].
[6]     Clinicaltrials.gov. Tezepelumab COPD Exacerbation Study (COURSE) [Online]. Available at: https://clinicaltrials.gov/ct2/show/NCT04039113. [Last accessed: October 2021].
[7]     Clinicaltrials.gov. Efficacy and Safety of Tezepelumab in Participants With Severe Chronic Rhinosinusitis With Nasal Polyposis (WAYPOINT). Available at: https://clinicaltrials.gov/ct2/show/NCT04851964. [Last accessed: October 2021].