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Onpattro
Published
Product name
Onpattro
Active ingredient
Patisiran
Submission type
New chemical entity
Decision
Approved
Decision date
Registration date
What this medicine was approved for
Onpattro (patisiran) was approved for the following therapeutic use:
Onpattro is indicated for the treatment of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) in adult patients with stage 1 or stage 2 polyneuropathy.
How this medicine works
Onpattro contains patisiran, a double stranded small interfering ribonucleic acid (siRNA) that targets a genetically conserved sequence in the 3’ untranslated region of a number of variant and wild-type transthyretin messenger ribonucleic acid (TTR mRNA). Patisiran is formulated as lipid nanoparticles to deliver the siRNA to hepatocytes, the primary source of TTR protein in the circulation. Through a process called RNA interference (RNAi), patisiran causes the catalytic degradation of TTR mRNA in the liver, resulting in a reduction of serum TTR protein.
Why the TGA approved or did not approve this medicine
The decision was based on quality (chemistry and manufacturing), nonclinical (pharmacology and toxicology), clinical (pharmacology, safety and efficacy) and risk management plan information submitted by the sponsor. The benefit-risk profile of Onpattro was considered favourable for the therapeutic use approved.
Sponsor