NICE recommends Onpattro to treat hereditary transthyretin-mediated amyloidosis

The National Institute for Health and Care Excellence (NICE) has recommended the use of Alnylam’s Onpattro (patisiran), after issuing a preliminary ‘no’, back in December 2018. 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. This recommendation 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 NICE in early 2019, putting Ionis ahead in the running.