Results from a global open-label extension study evaluating the long-term effects of Alnylam Pharmaceuticals’ (ALNY -2.4%) ONPATTRO (patisiran) in polyneuropathy of hereditary ATTR (hATTR) amyloidosis showed a sustained treatment benefit. The data were presented at the Peripheral Nerve Society Annual Meeting in Genoa, Italy.
At month 6, serum transthyretin levels dropped ~80% in patients in the placebo arm who switched to patisiran and were maintained over time. Patients on treatment for 30 – 36 months experienced sustained improvements in neuropathy impairment and quality-of-life (QOL) measures versus baseline.
A new analysis of the Phase 3 APOLLO study showed that patients who were previously treated with Pfizer’s (PFE -0.2%) tafamidis (branded as Vyndaqel and Vyndamax) and received patisiran for 18 months showed significant improvements in polyneuropathy and QOL measures, on par with the overall study population.
The company also announced an indirect comparison between ONPATTRO and Ionis Pharmaceuticals (IONS -1.7%) and Akcea Therapeutics’ (AKCA-1.5%) TEGSEDI (inotersen) that it says showed “favorable treatment effects” of patisiran.
The FDA approved ONPATTRO in August 2018 for the indication.
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