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Orphan Drug Designation for AstraZeneca’s Fasenra

AstraZeneca scientist

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Fasenra (benralizumab) for the treatment of hypereosinophilic syndrome (HES).

HES is a group of rare, potentially fatal disorders characterised by high numbers of eosinophils in blood and tissues, which can cause progressive damage to any organ in the body. The FDA grants ODD status to medicines intended for the treatment, diagnosis or prevention of rare diseases or disorders that affect fewer than 200,000 people in the US.

Phase II clinical trial

A Phase II clinical trial of Fasenra for the treatment of HES has been conducted by the United States National Institutes of Health in collaboration with AstraZeneca, with results expected to be published later in 2019. In the trial, Fasenra depleted blood eosinophils at week 12 compared with placebo, the primary endpoint of the trial, with evidence of eosinophil clearance in affected tissue at week 24.

“In patients with hypereosinophilic syndrome, high levels of eosinophils contribute to a range of debilitating symptoms and can even lead to life-threatening organ damage. Based on results from the Phase II trial, we believe Fasenra has the potential to address critical unmet medical needs in patients living with hypereosinophilic syndrome,” says Mene Pangalos, Executive Vice President, R&D BioPharmaceuticals, Astrazeneca.

First respiratory biologic

Fasenra is AstraZeneca’s first respiratory biologic and is currently approved as an add-on maintenance treatment for severe, eosinophilic asthma in the US, EU, Japan and several other countries. In November 2018, the FDA granted ODD for Fasenra for the treatment of eosinophilic granulomatosis with polyangiitis (EGPA).