Akcea obtains NICE approval for stage 1 or 2 polyneuropathy in adult patients with hATTR

Akcea Therapeutics’ Inotersen has received recommendation from NICE, meaning the therapy will now be made available to NHS patients in England and Wales for the treatment of Stage 1 or Stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). 

hATTR is a serious condition caused by “the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits” in the body’s tissues, including in the heart, peripheral nerves and intestinal tract. As the disease progresses, peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy often manifest, with patients living for only a median of 4.7 years following diagnosis.

As per the NHS authorisation, the therapy will now be available to patients via the health service in England within 90 days of the recommendation, while Welsh patients will have access earlier due to a commitment by the NHS in Wales to having funding available earlier.

“This is a great milestone for inotersen and marks the successful conclusion of this health technology assessment for inotersen,” commented Dr Richard A Jones, SVP Head of Europe for Akcea Therapeutics. “Patients in the UK with hATTR amyloidosis have to date had very limited treatment options to date, so this news will be well received among the amyloidosis community. Akcea are committed to advancing and making accessible transformative treatments for patients living with serious and rare diseases.”

Source: Pharmafile