Tofersen for treating amyotrophic lateral sclerosis caused by SOD1 gene mutations [ID3767]
In development
Reference number: GID-HST10062
Expected publication date: TBC
Following a challenge received from the company in September 2023, NICE sought further clinical opinion regarding the topic routing. We have concluded that Tofersen for SOD1-ALS fails to meet HST criterion 1 “The disease is very rare defined by 1:50,000 in England, and the topic continues to be routed to the Technology Appraisal (TA) programme."
Please note that following on from advice received from the company, further information regarding the timelines for this appraisal will be available in due course.
In the meantime, NICE will continue to monitor the topic and will provide an update as and when the situation changes.