patisiran

This page shows the latest patisiran news and features for those working in and with pharma, biotech and healthcare.

Akcea steals march on Alnylam with NICE approval

The draft decision from NICE’s highly specialised technologies (HST) workstream is a major boost for Akcea, which was initially rejected in December alongside its close rival, Alnylam’s Onpattro (patisiran).

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Orphan drugs dominate FDA's record-breaking year

Other highlights of the year included Alnylam’s hereditary ATTR amyloidosis drug Onpattro (patisiran) becaming the first RNA interference (RNAi) therapy to get a green light from the FDA.
NICE says no to rival Alnylam and Akcea amyliodsis drugs – but deal could be done

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 ... Commenting on the draft
Alnylam gets EU nod for Onpattro amid pricing backlash

The EMA has cleared Onpattro (patisiran) – the first RNA interference drug approved in the EU – for adult hATTR amyloidosis patients with stage 1 or 2 nerve damage (polyneuropathy), one of the ... intravenous injection of patisiran every three
ICER says Alnylam’s Onpattro should cost 90%-95% less

In draft guidance, the Institute for Clinical and Economic Review (ICER) says that RNA interference drug Onpattro (patisiran) – and Akcea/Ionis’ antisense candidate Tegsedi (inotersen) which is due for an FDA
Alnylam claims RNAi milestone with Onpattro approval

Alnylam is however modelling an average $345, 000 annual cost after discounts, and is also offering a money back guarantee for insurers in the form of a rebate if patisiran ... John Maraganore has led Alnylam since 2002. Alnylam’s chief executive –

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Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe

Its pivotal APOLLO trial showed nearly all patisiran-treated patients saw the disease halted or slowed, with more than half seeing their neuropathy reversed.
Deal Watch table for January 2014

Alnylam/ Genzyme. Extended agreement. Additional markets for patisiran and 3 pipeline products with option to all rare disease products.
Pharma deals during January 2014

The original deal was forged back in 2012 when Genzyme gained access to patisiran for the treatment of transthyretin familial amyloid polyneuropathy in Japan and other Asian markets.

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