(CercleFinance.com) – Ionis Pharmaceuticals announced on Monday that the US Food and Drug Administration (FDA) has granted orphan drug designation to eplontersen, its treatment for hereditary transthyretin (ATTR) amyloidosis, a genetic disease progressive and potentially fatal.
FDA 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 United States.
Last December, Ionis signed a strategic collaboration with AstraZeneca for the development and commercialization of the product.
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