Patisiran (Onpattro)
Onpattro treats nerve damage caused by a rare inherited disease, hereditary ATTR amyloidosis. It wraps small strands of RNA in a lipid nanoparticle that carries them to the liver, where they switch off production of the faulty protein behind the disease.
BioPharm International, 13 Aug 2018How far from your life: You can get it now, though it may not be common yet.
Independently sourcedFrom lab to your life
Why it’s on step 4
- 1Lab discovery
- 2Working prototype
- 3Real-world testing
- 4Approved or on sale
- 5Everyday life
The rule
A regulator approved or cleared it where approval applies (the FDA, the EMA, a CE mark), or it is sold to the public or to customers beyond a trial.
- The FDA approved Onpattro, a first-of-its-kind RNA-based medicine, for nerve damage caused by hereditary ATTR amyloidosis in adults. BioPharm International, 13 Aug 2018
The short version
What you need to know
- Why it matters
By cutting the faulty protein, it can reduce the deposits that build up in nerves, improving symptoms.
BioPharm International, 13 Aug 2018- Is it really nano?
Truly nano
The lipid nanoparticle is what carries the RNA to the liver. The FDA notes that the carrier can also set off an immune response of its own.
US FDA, CDER science spotlight- Where you’ll find it
Given by infusion to adults with nerve damage from hereditary ATTR amyloidosis.
Alnylam, 10 Aug 2018- Who’s behind it
Alnylam Pharmaceuticals
On the record
Every step, dated
Newest first. Company-reported means the only source so far is the company itself.
- Approval
The FDA approved Onpattro, a first-of-its-kind RNA-based medicine, for nerve damage caused by hereditary ATTR amyloidosis in adults.
BioPharm International, 13 Aug 2018
Who makes it
The companies
- ALNYNasdaqAlnylam Pharmaceuticals
Makes Onpattro, an RNA medicine that reaches the liver inside a lipid nanoparticle.
We track:Onpattro
Sources
Where every line comes from
Each source with the passage, word for word, that backs what we wrote.
- BioPharm International, 13 Aug 2018
More specifically, Onpattro encases siRNA into a lipid nanoparticle that delivers the drug directly into the liver
- US FDA, CDER science spotlight
Patisiran is encased in a lipid nanoparticle, which carries and delivers the drug to the liver
- Alnylam, 10 Aug 2018
for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
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