AstraZeneca’s Wainzua recommended on the NHS in England

UK pharma major AstraZeneca (LSE: AZN) today announced that the National Institute for Health and Care Excellence (NICE) published its final draft guidance (FDG) recommending Wainzua (eplontersen) as a treatment option for hereditary transthyretin-related amyloidosis (ATTR) in adults with stage 1 or stage 2 polyneuropathy across England and Wales.

The recommendation by the NICE through its Proportionate Approach to Technology Appraisals (PATT) process, follows the recent marketing authorization from the Medicines and Healthcare products Regulatory Agency (MHRA) in this setting across Great Britain and has facilitated accelerated patient access to a patient population with a high unmet need. The European Medicines Agency’s human health committee, CHMP, recommended approval for a Wainzua earlier this month.

AstraZeneca noted that this NICE recommendation is based on results from the NEURO-TTRansform Phase III clinical trial which enrolled 168 patients (144 assigned to subcutaneous eplontersen) and included 60 historical placebo patients. At week 65, in a co-primary endpoint of the trial, eplontersen achieved a least squares (LS) mean reduction of 81.7% in serum TTR protein levels from baseline compared to an 11.2% reduction from baseline in the external placebo group (difference, -70.4% [95% CI, -75.2% to -65.7%]; p<0.001].2 Results showed that eplontersen reduced serum TTR protein levels as early as week 5.2