Akcea Therapeutics UK Ltd has announced that the National Institute for Health and Care Excellence (NICE) has published its final Highly Specialised Technologies (HST) Guidance for Tegsedi® (inotersen) for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).1,2
This follows the positive Final Evaluation Document (FED)3 recommending that patients in England with this rare, inherited, severely debilitating and fatal disease, can access the treatment on the NHS.
hATTR amyloidosis is a severe, progressive, and life-threatening disease caused by the abnormal formation of the TTR protein and aggregation of TTR amyloid deposits in various tissues and organs throughout the body, including in peripheral nerves, the heart and intestinal tract.4 The progressive accumulation of TTR amyloid deposits in these organs often leads to intractable peripheral sensorimotor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.2 hATTR amyloidosis causes significant morbidity and progressive decline in quality of life, severely impacting activities of daily living.2 The disease often progresses rapidly and can lead to premature death.2 The median survival is 4.7 years from diagnosis.5
Inotersen is an antisense oligonucleotide (ASO) inhibitor of human transthyretin (TTR) production. It is the first and only subcutaneous RNA-targeting drug designed to reduce the production of human transthyretin (TTR) protein.6
This HST Guidance is the final recommendation from NICE regarding how this treatment should be used in the NHS in England. Now that the final guidance has been published, the NHS mandate requires that inotersen is available for routine use within 90 days for patients in England. The NHS in Wales has committed to have funding available earlier, 60 days post-FED.
“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.”
- National Institute for Health and Care Excellence (NICE). Tegsedi (inotersen) Highly Specialised Technology Guidance (HST9) Available at: https://www.nice.org.uk/guidance/hst9. Accessed 22 May 2019.
- National Institute for Health and Care Excellence (NICE). Tegsedi (inotersen) Highly Specialised Technology Guidance (HST9). Final Guidance Document Available at https://www.nice.org.uk/guidance/hst9/resources/inotersen-for-treating-h… Accessed 22 May 2019.
- National Institute for Health and Care Excellence (NICE). Tegsedi (inotersen) Final Evaluation Document. Available at: https://www.nice.org.uk/guidance/hst9/documents/final-evaluation-determi… . Last accessed May 2019.
- Damy T et al. (2015) Cardiac Findings and Events Observed in an Open-Label Clinical Trial of Tafamidis in Patients with non-Val30Met and non-Val122Ile Hereditary Transthyretin Amyloidosis. J Cardiovasc Transl Res. 8(2):117-127.
- Swiecicki PL et al. Hereditary ATTR amyloidosis: a single-institution experience with 266 patients. Amyloid 2015;22(2):123-3.
- eMC. Tegsedi Summary of Product Characteristics. Available at: www.medicines.org.uk/emc/product/10011/smpc. Last accessed May 2019.