AstraZeneca: orphan drug designation for Ionis

(CercleFinance.com) - Ionis Pharmaceuticals announced on Monday that the US FDA has granted orphan drug designation to eplontersen, its treatment for hereditary transthyretin amyloidosis (HTRA), a progressive and potentially fatal genetic disease.


The FDA's orphan drug designation is intended to facilitate the emergence of drugs for the treatment of relatively rare diseases, affecting fewer than 200,000 people in the US.

Last December, Ionis signed a strategic collaboration with AstraZeneca for the development and commercialisation of the product.


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