(News Bulletin 247) – AstraZeneca and its American partner Ionis announced on Monday new positive phase III data relating to eplontersen, a treatment for hereditary transthyretin amyloid neuropathy (HAT-TTR), a disease likely to lead to the death of patients. .
According to detailed data presented today at the American Academy of Neurology (AAN) meeting in Boston, the 66-week trial met all of its primary and secondary endpoints compared to a placebo.
The treatment demonstrated both an improvement in terms of transthyretin concentration in the blood, functional impairment and quality of life.
NAH-TTR, which causes peripheral nerve damage, results in motor disability within five years of diagnosis and, if left untreated, death within ten years.
According to AstraZeneca and Ionis, these data support the registration application filed last month in the United States, which should soon be followed by the filing of a file in Europe.
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