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hATTR

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

Alnylam gains key NICE approval for Onpattro

Alnylam gains key NICE approval for Onpattro

Both Onpattro and Tegsedi are licensed to treat the ultra-rare disease hereditary transthyretin-mediated amyloidosis (hATTR) in adults with stage 1 or 2 polyneuropathy, or damage to peripheral nerves. ... Gene-silencing is a promising area of medicine

Latest news

  • Speedy approval for Pfizer's Onpattro rival Speedy approval for Pfizer's Onpattro rival

    Alnylam's Onpattro is indicated for a different disease ATTR subtype,  hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) where patients suffer polyneuropathy complications from the condition. ... Despite the different indications,

  • Akcea steals march on Alnylam with NICE approval Akcea steals march on Alnylam with NICE approval

    While the drugs represent a major step forward in improving the outlook for patients with hATTR, they do represent a major expense for healthcare systems, as they require lifelong treatment. ... Richard A. Jones,  SVP Head of Europe for Akcea

  • Sanofi exits R&D pact with Alnylam on rare disease Sanofi exits R&D pact with Alnylam on rare disease

    Genzyme had previously licensed rights in 2012 to Alnylam’s lead RNAI drug Onpattro (patisiran) for hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis), which became the first drug in the RNAi class ... returning development and

  • NICE says no to rival Alnylam and Akcea amyliodsis drugs – but deal could be done 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 NICE has issued draft guidance not recommending two new rival treatments for the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR) – Alnylam’s Onpattro and ... Just 150 people in

  • Alnylam gets EU nod for Onpattro amid pricing backlash Alnylam gets EU nod for Onpattro amid pricing backlash

    for tafamidis as a treatment for cardiomyopathy associated with hATTR amyloidosis after reporting positive results in the phase III ATTR-ACT trial. ... Until recently, patients diagnosed with hATTR amyloidosis faced an uncertain future,” says Professor

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