Patisiran

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Patisiran
Patisiran.png
Clinical data
Trade namesOnpattro
Other namesALN-18328
ATC code
Identifiers
CAS Number
DrugBank
UNII
KEGG
Chemical and physical data
FormulaC412H520N148O290P40
Molar mass13424.388 g·mol−1

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.[1]

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.[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.

History[edit]

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.[2][3] It was approved by the FDA in August 2018 and is expected to cost around $345,000 to $450,000 per year.[4]

References[edit]

  1. ^ a b Loftus, Peter (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". Wall Street Journal. Retrieved 10 August 2018.
  2. ^ "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.
  3. ^ Brooks, Megan (10 August 2018). "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". Medscape. WebMD. Retrieved 10 August 2018.
  4. ^ Lipschultz, Bailey; Cortez, Michelle (10 August 2018). "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". Bloomberg. Retrieved 11 August 2018.