AstraZeneca and Amgen secure FDA breakthrough designation for asthma drug tezepelumab

In a significant advancement for asthma treatment, AstraZeneca, in partnership with Amgen, has received the coveted breakthrough therapy designation from the US Food and Drug Administration (FDA) for their innovative asthma drug, tezepelumab. This designation is specifically for the treatment of patients with severe asthma who do not exhibit an eosinophilic phenotype and are being treated with a combination of inhaled corticosteroids and long-acting beta2-agonists, with or without oral corticosteroids and other asthma controllers.

Tezepelumab has shown potential far beyond the current biologic therapies available for asthma, prompting high hopes for its ability to treat a broader spectrum of severe asthma cases. The drug works by targeting and blocking thymic stromal lymphopoietin (TSLP), an epithelial cytokine critical in initiating and sustaining airway inflammation. By inhibiting TSLP, tezepelumab may prevent the release of pro-inflammatory cytokines by immune cells, potentially reducing asthma exacerbations and enhancing overall asthma control.

The FDA’s decision was heavily influenced by the results from the PATHWAY phase 2b trial, which indicated that tezepelumab significantly reduced the annual asthma exacerbation rate compared to placebo across a diverse group of severe asthma patients, regardless of their Type 2 (T2) biomarker status. This broad efficacy highlights tezepelumab’s unique position in the severe asthma treatment landscape, capable of addressing needs unmet by existing therapies.

Sean Bohen, Executive Vice President of Global Medicines Development and Chief Medical Officer at AstraZeneca, expressed enthusiasm about the breakthrough designation, noting that it will expedite the drug’s development and availability to patients. Similarly, David M. Reese, Executive Vice President of Research and Development at Amgen, emphasized the drug’s promise shown in the PATHWAY trial and the potential to rapidly bring this new therapy to patients in need thanks to the FDA’s designation.

This breakthrough therapy designation marks a pivotal milestone not only for AstraZeneca and Amgen but also for the field of respiratory medicine. It is AstraZeneca’s seventh breakthrough designation from the FDA since 2014 and its first in the respiratory domain. With tezepelumab now progressing through the phase 3 PATHFINDER trial, both the medical community and patients are eagerly anticipating its potential impact on managing severe asthma more effectively.

The designation is particularly significant as it recognizes tezepelumab’s potential to change the treatment paradigm for severe asthma—a condition that affects millions worldwide and poses substantial health burdens. The ongoing phase 3 trials will further elucidate the drug’s benefits and safety profile, potentially leading to a new standard of care for asthma patients.