AZ gets approval for drug targeting rare disease – it may reduce cortisone dependence
Av Eosinophilic granulomatosis with polyangiitis (EGPA), formerly known as Churg-Strauss syndrome, is characterised by an increased number of eosinophilic granulocytes in organs and in the bloodstream, causing inflammation of small and medium-sized blood vessels. The disease invariably causes lung damage, and often affects other organs such as the peripheral nerves, skin, heart, stomach and kidneys. When left untreated, the disease can be fatal.
Currently, the disease is mainly treated with high doses of cortisone combined with immunosuppressive drugs. However, an additional treatment is now being introduced that may help a large proportion of patients.
The drug is AstraZeneca’s Fasenra, which has already been approved for severe eosinophilic asthma and is now approved for wider use. In a study on which the approval is based, nearly 60% of patients achieved remission and over 41% no longer needed to take oral corticosteroids such as cortisone.
He also stresses that corticosteroids can cause severe and long-lasting side effects.
The active substance in Fasenra is the monoclonal antibody benralizumab. The medicine is now also being tested for several other indications and has been granted orphan drug designation for development against eosinophilic esophagitis, an inflammation characterised by eosinophilic granulocytes in the oesophagus.
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