Alnylam gains EU approval for RNAi therapy Onpattro to treat hATTR

Alnylam Pharmaceuticals announced Thursday that the European Commission granted marketing authorisation for Onpattro (patisiran) to treat hereditary transthyretin-mediated (hATTR) amyloidosis in adults with stage 1 or 2 polyneuropathy. The company noted that the decision, which follows FDA clearance earlier this month, makes Onpattro the first RNAi therapy to be approved in the EU. 

The latest approval follows a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use issued in July. The European filing was backed by data from the Phase III APOLLO trial, in which Onpattro was linked to significant improvements in various symptoms from baseline compared to placebo as measured by the modified Neuropathy Impairment Score +7, as well as improvements in quality of life. 

Earlier this week, the Institute for Clinical and Economic Review (ICER) released a report suggesting that the $345 000 annual net price of Onpattro should be reduced by 90 percent to 95 percent. "The announced price of [Onpattro], even taking into account expected discounts, far exceeds commonly cited cost-effectiveness thresholds," remarked ICER chief scientific officer Dan Ollendorf. Alnylam has agreed in principle to the structure and key terms of "value-based agreements" with certain insurers, but has provided few details on the discounts.

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Meanwhile, shares in Alnylam have jumped more than 20 percent since Pfizer unveiled data this week from the late-stage ATTR-ACT study, showing that its investigational therapy tafamidis cut the risk of mortality by 30 percent in patients with wild-type or hereditary transthyretin amyloid cardiomyopathy, versus placebo. Jefferies analyst Maury Raycroft suggested "some weaknesses" in the ATTR-ACT results included that the benefit of tafamidis on hATTR patients was not clear, and this "ultimately should enhance [Onpattro's] commercial opportunity," while the impact of Pfizer's drug on Ionis Pharmaceuticals and Akcea Therapeutics' Tegsedi (inotersen) may also be limited. Tegsedi gained European approval in July for stage 1 or 2 polyneuropathy in adults with hATTR, while the FDA is expected to issue a final decision in October.

For related analysis, see ViewPoints: Alnylam rides high on Pfizer's success. See also ViewPoints: Conservative label, aggressive price for Alnylam's Onpattro.

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