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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

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

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

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

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

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 NICE has issued draft guidance not recommending two new rival treatments for the rare inherited condition hereditary transthyretin-related amyliodsis (hATTR) – Alnylam’s Onpattro and ...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

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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20 for 2020 – Five pharma leaders to watch in 2020

Onpattro was approved to treat adults with the. destruction of nerves that results from a rare disease, hereditary transthyretin- mediated (hATTR) amyloidosis, and was launched with a $450, 000 per year ... Standing in its way is a similarly priced
Alnylam’s big moment: bringing groundbreaking RNAi drugs to Europe

The drug is licensed to treat to adults with stage 1 or 2 polyneuropathy, the destruction of nerves that results from hereditary transthyretin- mediated (hATTR) amyloidosis, a rare, rapidly progressive, fatal ... Neurologists, cardiologists and

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