Patisiran

Patisiran, sold under the brand 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.[1]

Patisiran
Clinical data
Trade namesOnpattro
Other namesALN-18328
AHFS/Drugs.comMonograph
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC412H520N148O290P40
Molar mass13424.388 g·mol−1

It is the first small interfering RNA-based drug approved by the U.S. Food and Drug Administration (FDA) and the first drug approved by the FDA to treat this condition.[1] 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. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[2]

History

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.[3][4] It was approved for medical use in the United States and in the European Union in August 2018.[5][6] It is expected to cost around US$345,000 to US$450,000 per year.[7]

References
  1. Loftus P (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. Retrieved 10 August 2018.
  2. New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
  3. "FDA approves first-of-its kind targeted RNA-based therapy to treat a rare disease" (Press release). U.S. Food and Drug Administration (FDA). 10 August 2018. Retrieved 11 August 2018.
  4. Brooks M (10 August 2018). "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". Medscape. WebMD. Retrieved 10 August 2018.
  5. "Drug Approval Package: Onpattro (patisiran)". U.S. Food and Drug Administration (FDA). 7 September 2018. Retrieved 2 September 2020.
  6. "Onpattro EPAR". European Medicines Agency (EMA). Retrieved 2 September 2020.
  7. Lipschultz B, Cortez M (10 August 2018). "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". Bloomberg. Retrieved 11 August 2018.
  • "Patisiran". Drug Information Portal. U.S. National Library of Medicine.
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