The treatment is for a progressive, life-threatening disease called hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), which up until now was an area of significant unmet need in England.
The drug will now provide eligible patients suffering from the disease with a treatment option that addresses its underlying cause by reducing the production of an abnormal protein that damages nerves and organs across the body.
The drug’s journey has been parallel to Ionis’ Tegsedi, as they were both approved in Europe in 2018, but both rejected by NICE later in the year. Tegsedi was then approved by the organisation in early 2019, putting Ionis ahead in the running…
