Patisiran (brand name Onpattro) is a prescription medication used to treat the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is the first and only approved RNA interference (RNAi) therapeutic, and is administered via injection into a vein (intravenously). Patisiran works by targeting and silencing a mutated transthyretin gene responsible for hATTR amyloidosis, reducing production of the abnormal protein that builds up in the body and causes the disease.
Patisiran, sold under the brand name Onpattro, is a medication specifically designed to treat a rare disease called hereditary transthyretin-mediated amyloidosis (hATTR). Here's a breakdown of its key features:
Condition Treated:
- hATTR is a genetic condition where an abnormal form of a protein called transthyretin (TTR) builds up in various organs, particularly the nerves and heart, leading to damage and organ dysfunction.
How Patisiran Works:
- Patisiran is a first-of-its-kind small interfering RNA (siRNA) medication.
- siRNA molecules target specific genes and essentially silence them, preventing the production of certain proteins.
- In this case, Patisiran targets the TTR gene in the liver, where TTR is primarily produced. By reducing TTR production, Patisiran helps slow down the buildup of the abnormal protein and potentially alleviate symptoms.
Administration:
- Patisiran is administered intravenously (through a vein) by a healthcare professional in a hospital or clinic setting.
Benefits:
- Patisiran is the first FDA-approved treatment for hATTR and has shown effectiveness in clinical trials, particularly in improving nerve function and reducing symptoms like neuropathy (nerve pain, tingling, numbness).
Important Considerations:
- Patisiran is a complex medication and should only be used under the supervision of a doctor experienced in treating hATTR.
- It can cause side effects like infusion-related reactions (fever, chills, nausea) and reduced vitamin A levels. These can usually be managed by medical professionals.
- Patisiran is not a cure for hATTR, but it can be a valuable tool in managing the disease and improving quality of life for patients.