Patisiran

Patisiran
Clinical data
Trade names	Onpattro
Other names	ALN-18328
ATC code	N07XX12 (WHO) ;
Identifiers
CAS Number	1420706-45-1;
DrugBank	DB14582;
UNII	50FKX8CB2Y;
KEGG	D11116;
Chemical and physical data
Formula	C412H520N148O290P40
Molar mass	13424.388 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

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

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.

This article is issued from Wikipedia. The text is licensed under Creative Commons - Attribution - Sharealike. Additional terms may apply for the media files.

[Loftus_2018-1] 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.