AstraZeneca and Ionis eye US approval after positive trial data
Based on the results, the companies plan to file an application to market the therapy in the US later this year in patients with hereditary transthyretin-mediated amyloid polyneuropathy
An interim analysis of AstraZeneca and partner Ionis Pharma’s eplontersen showed the experimental drug met the main goals in a late-stage trial in patients with a rare, fatal disease, the Anglo-Swedish drugmaker said yesterday.
Based on the results, the companies plan to file an application to market the therapy in the US later this year in patients with hereditary transthyretin-mediated amyloid polyneuropathy.
The disease – which affects an estimated 40,000 patients globally – leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade, according to AstraZeneca.
The Cambridge, UK-based company agreed to pay California-based Ionis $200 million upfront, with up to $485 million in conditional payments following regulatory approvals as well as up to $2.9 billion in sales-related milestone payments in a deal to jointly develop and commercialise the drug last year.
Edits by EP News Bureau
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