NICE has issued draft guidance not recommending two new rival treatments for the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR) – Alnylam’s Onpattro and Tegsedi from Akcea (a subsidiary of Ionis).
The draft highly specialised technologies guidance on Tegsedi (inotersen) and Onpattro (patisiran) concluded that both treatments slows progression of the disease and improve quality of life, but says uncertainty around these benefits being maintained in the longer-term, as well as uncertainties around economic modelling for both treatments, means its first ruling is a ‘no’.
Gaining NICE recommendation would be a major milestone for both companies – Onpattro is Alnylam’s debut product from its RNA interference (RNAi) drug platform, while Tegsedi is similarly Akcea’s first product from its antisense drug technology, which also targets RNA.
Onpattro (top) and Tegsedi could gain recommendation via further negotiation
Both companies have already offered confidential discounts on their drugs, but the NICE committee has rejected these, and says it didn’t think managed access schemes would resolve the cost effectives uncertainties.
However, an initial rejection followed by further negotiation on data and price has become standard practice in NICE appraisals, and both firms have a chance to secure recommendation if they make further concessions.
Just 150 people in England are thought to be affected by hATTR, an ultra-rare progressive condition that causes the liver to produce abnormal transthyretin protein, which can build up in the body and cause polyneuropathy and cardiomyopathy which shorten a patient’s life.
Onpattro works by stopping the production of transthyretin by the liver (including abnormal transthyretin). It is given by intravenous infusion every three weeks at a dose of 0.3 mg/kg. It costs £7,676.45 per 10-mg vial and is a lifelong treatment.
Like its rival, Tegsedi is a life-long treatment and works by suppressing the production of transthyretin. It can be self-administered once weekly by injection and costs £5,920 per 284 mg weekly dose.
Commenting on the draft guidance, Brendan Martin, general manager of Alnylam UK & Ireland said: “Patisiran is breaking new ground, offering eligible people with hATTR amyloidosis a new treatment option with the potential to help improve debilitating neuropathy symptoms.”
He added that the novel, highly-specialised nature of Onpattro “makes it challenging to assess its full value” but sounded confident an agreement could be reached.
“Our focus now will be on addressing the immediate questions posed by the Committee and on working towards securing a positive, sustainable agreement that represents value for the NHS and that would help transform the lives of people living with this terrible disease.”
The consultation on the draft guidance on both treatments is open until 9 January 2019.