What is Onpattro (patisiran)?
Onpattro (patisiran) is indicated for the treatment of stage 1 or stage 21 polyneuropathy2 in adult patients with hereditary transthyretin amyloidosis (hATTR)1,2.
Polyneuropathies, or peripheral neuropathies, result from damage to the peripheral nerves (nerves that go from the spinal cord to the arms, hands, legs, and feet). This degeneration of the neurons is progressive and causes weakness, numbness and pain in the extremities4.
How does Onpattro (patisiran) work?
Transthyretin amyloidosis is a rare disease characterised by the presence of a defective blood protein called transthyretin (TTR)7. This defective protein breaks easily, which results in the deposition of a fibrous substance (amyloid fibrils) within the body7. The accumulation of amyloid fibrils causes damage to body structures, like tissues and organs, and prevents their normal function7. Symptoms and signs can differ depending on the location of the amyloid protein deposits5. When the nerves are affected the disease is called hereditary transthyretin amyloidosis (hATTR)8.
Patisiran is the first of a new class of targeted RNA-based therapies interfering with the production of mutant transthyretin (TTR) protein3,6. In this way, it reduces the formation of amyloids and relieves the symptoms of polyneuropathy associated with hATTR amyloidosis6.
Where has Onpattro (patisiran) been approved?
Onpattro (patisiran) was approved for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR)1,2 by:
Food and Drug Administration (FDA), USA, October 6, 20181
European Medical Agency (EMA), European Union, August 27, 20182.
Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.
How is Onpattro (patisiran) taken?
The recommended Onpattro (patisiran) dosage is based on body weight1,2:
For patients weighing less than 100 kg, 0.3 mg/kg as intravenous infusion once every 3 weeks
For patients weighing more than 100 kg, 30 mg as intravenous infusion once every 3 weeks.
All patients should receive premedication prior to Onpattro administration to reduce the risk of
infusion-related reactions1. The usual premedication should be given at least 60 minutes prior to the start of infusion1.
Complete information about Onpattro (patisiran) dosage and administration can be found in the official prescribing information listed in our resources section1,2.
Note: Please consult with your treating doctor for personalised dosing.
Are there any known side effects or adverse reactions of Onpattro (patisiran)?
Common adverse reactions
The most common adverse reactions listed in the prescribing information include1,2:
upper respiratory tract infections
infusion-related reactions such as:
stomach pain
feeling sick
body aches
headache
fatigue.
For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information1,2.
