News

January 2019:

If you are interested in being a part of the ATTR
Amyloidosis Patients’ Association, please complete the contact form.

December 2018:

The NICE assessments of Patisiran and Inotersen have been made public on their website. These recommendations are not final guidance and may change after consultation.  The UK ATTR Amyloidosis Patients Association is involved in the NICE process. Our summary of the significance of these assessments is as follows:

NICE assessment of Patisiran.

The National Institute for Health and Care Excellence (NICE) has published an Evaluation Consultation Document (ECD) on its assessment of patisiran. It states that “Patisiran reduces disability, increases quality of life and is innovative”. However, there remain some questions about its cost-effectiveness, therefore NICE state that “Patisiran is not recommended, within its marketing authorisation, for treating hereditary transthyretin-related amyloidosis in adults”.
It is important to keep in mind that this is an initial draft guidance and further discussions regarding the cost effectiveness of the drug will be held. UK ATTR Patient Association expects that NICE, Alnylam and NHS England will continue to work towards making patisiran available to patients in England and Wales. A final decision is anticipated in the new year.
Patisiran (Onpattro®) is a ribonucleic acid interference agent that suppresses transthyretin (TTR) production by the liver (including abnormal TTR). It is administered once every 3 weeks by intravenous infusion at a dose of 0.3 mg/kg. It has a marketing authorisation in the UK for treating ‘hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy’.

NICE assessment of Inotersen.

The National Institute for Health and Care Excellence (NICE) has published an Evaluation Consultation Document (ECD) on its assessment of Inotersen. It states that “Inotersen slows progression of the disease considerably and is innovative”. However, there remain some questions about its cost-effectiveness, therefore NICE state that “Inotersen is not recommended, within its marketing authorisation, for treating polyneuropathy in adults with hereditary transthyretin-related amyloidosis”.
It is important to keep in mind that this is an initial draft guidance and further discussions regarding the cost effectiveness of the drug will be held. UK ATTR Patient Association expects that NICE, Akcea and NHS England will continue to work towards making Inotersen available to patients in England and Wales. A final decision is anticipated in the new year.
Inotersen (Tegsedi, Akcea Therapeutics) is a novel, first-in-class 2’-O-2- methoxyethyl phosphorothioate antisense oligonucleotide that inhibits production of transthyretin (TTR) in adults with hereditary transthyretinrelated (hATTR) amyloidosis. Inotersen has a marketing authorisation for the treatment of stage 1 or stage 2 polyneuropathy in adults with hATTR amyloidosis

September 2018:

Recent press coverage of ATTR amyloidosis and treatment advances:

– BBC News
– iNews

The National Amyloidosis Centre is holding an ATTR Amyloidosis Patient and Family Infoday at the Royal Free Hospital on Friday 25 January 2019. The Infoday will be held in the Royal Free Atrium 10am-3pm. See flyer below for more details:

ATTR InfoDay Advert