The generic name for Onpattro is patisiran.
Onpattro is a well-known brand name for a specialized medication designed to address specific neurological conditions. Understanding both the brand and generic names helps patients, caregivers, and healthcare professionals identify and discuss medications accurately.
Understanding Onpattro (Patisiran)
Patisiran, marketed under the brand name Onpattro, represents a significant advancement in the treatment of a rare and progressive disease. It is an innovative medication that utilizes a unique mechanism of action to target the root cause of certain conditions.
Here's a quick overview:
| Aspect | Detail |
|---|---|
| Brand Name | Onpattro |
| Generic Name | Patisiran |
| Drug Class | Transthyretin-directed small interfering RNA (siRNA) |
| Formulation | Injectable |
| Primary Use | Treatment for polyneuropathy (multiple nerve damage) caused by hereditary transthyretin-mediated amyloidosis (ATTR) in adults. |
How Patisiran Works
Patisiran is classified as an injectable transthyretin-directed small interfering RNA (siRNA). This means it works by specifically targeting and reducing the production of abnormal transthyretin (TTR) protein in the liver. In hereditary ATTR amyloidosis, mutations in the TTR gene lead to the production of misfolded TTR proteins. These misfolded proteins accumulate as amyloid deposits in various organs, including the nerves, causing damage and leading to polyneuropathy.
By silencing the specific messenger RNA (mRNA) responsible for producing this abnormal TTR protein, patisiran effectively lowers the levels of TTR in the bloodstream. This action helps to reduce the further buildup of amyloid deposits and can improve or stabilize nerve function in affected individuals.
Therapeutic Importance
The development of patisiran has provided a crucial treatment option for adults suffering from polyneuropathy associated with hereditary transthyretin-mediated amyloidosis. Prior to such targeted therapies, treatment options for this condition were limited, often focusing on managing symptoms rather than addressing the underlying cause.
Key benefits of a specific therapy like patisiran include:
- Targeted Action: Directly addresses the production of the harmful protein.
- Disease Modification: Aims to slow the progression of nerve damage rather than just alleviate symptoms.
- Improved Quality of Life: Can lead to improvements in nerve function, reducing pain, weakness, and other debilitating symptoms of polyneuropathy.
Understanding the generic name "patisiran" in conjunction with its brand name "Onpattro" is vital for effective communication in healthcare and for comprehending the specific nature of this advanced therapeutic agent.
