NICE backs Akcea’s Tegsedi, pipping Alnylam to hATTR okay

NICE has recommended that Akcea’s hereditary transthyretin-related amyloidosis (hATTR) treatment Tegsedi be made available on the NHS in England, ahead of a rival therapy from Alnylam. Both Tegsedi (inotersen) and Alnylam’s rival hATTR therapy Onpattro (patisiran) were turned down by the UK healthy technology assessment (HTA) agency in draft guidance published in December on the grounds that their cost was too high to balance their clinical benefit. At full price both drugs cost around £300,000 per year per patient, but there are only around 150 patients with hATTR in the UK, according to NICE. Carlos Heras-Palou of the UK ATTR Amyloidosis Patients Association said that NICE’s decision marks “a landmark day for people with hATTR amyloidosis who have had very limited options available to them to date.” “There is a critical need for innovative new therapies for people across the UK living with this debilitating disease. We hope inotersen will be available to patients in the UK very soon,” he added. After a confidential price cut, Akcea has persuaded NICE to change its position on Tegsedi, paving the way for the first-ever treatment for hATTR to be made available patients with the ultra-rare, progressive condition, which causes the liver to produce abnormal transthyretin protein.

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