UK – NICE says no to rival Alnylam and Akcea amyliodsis drugs – but deal could be done

Room for manoeuvre after initial rejection. NICE has issued draft guidance not recommending two new rival treatments for the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR) – Alnylam’s Onpattro and Tegsedi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.

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…