A gene-silencing therapy for a rare hereditary condition has been approved for NHS use in England.
Patisiran 鈥� marketed under the brand name Onpattro 鈥� targets a faulty gene in the liver that causes hereditary transthyretin-mediated amyloidosis.
This condition can be inherited from either parent and causes sticky amyloid protein to build up in organs and around nerves. This can lead to problems with limb movements, vision and heart function, and can also trigger chronic burning neuropathic pain. The condition is thought to affect about 100 people in the UK.
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Patisiran works by using genetic material called RNA to block the action of the faulty gene. It was approved by the US Food and Drugs Administration last year.
鈥淧atisiran has shown in its main clinical study that it can halt or even improve potentially debilitating symptoms of this disease in the majority of patients,鈥� says Philip Hawkins, head of the National Amyloidosis Centre at London鈥檚 Royal Free Hospital. 鈥淭his means we now have a real possibility of preserving quality of life for eligible patients for longer than has so far been possible.鈥�
The drug is administered once every three weeks by intravenous infusion and can cost as much as 拢300,000 a year per patient. According to NICE, the company has a 鈥渃ommercial arrangement鈥� which 鈥渕akes patisiran available to the NHS with a discount鈥� 鈥� the size of which is confidential.
