|Chemical and physical data|
|Molar mass||424.388 g·mol−1 13|
Patisiran (trade name Onpattro) is a medication for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis. Hereditary transthyretin-mediated amyloidosis is a fatal rare disease that is estimated to affect 50,000 people worldwide.
It is the first small interfering RNA-based drug approved by the FDA and the first drug approved by the FDA to treat this condition. It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.
Patisiran was developed and is marketed by Alnylam.
Patisiran was granted orphan drug status, fast track designation, priority review and breakthrough therapy designation due to its novel mechanism and the rarity of the condition it treats. It was approved by the FDA in August 2018 and is expected to cost around $345,000 to $450,000 per year.
- Loftus, Peter (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". Wall Street Journal. Retrieved 10 August 2018.
- "FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease" (Press release). U.S. Food and Drug Administration. 10 August 2018. Retrieved 11 August 2018.
- Brooks, Megan (10 August 2018). "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". Medscape. WebMD. Retrieved 10 August 2018.
- Lipschultz, Bailey; Cortez, Michelle (10 August 2018). "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". Bloomberg. Retrieved 11 August 2018.
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