Patisiran
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| Clinical data | |
|---|---|
| Trade names | Onpattro |
| Other names | ALN-18328 |
| AHFS/Drugs.com | Monograph |
| License data | |
| Routes of administration | Intravenous |
| ATC code | |
| Legal status | |
| Legal status |
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| Identifiers | |
| CAS Number | |
| DrugBank | |
| UNII | |
| KEGG | |
| Chemical and physical data | |
| Formula | C412H520N148O290P40 |
| Molar mass | 13424.388 g·mol−1 |
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]
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.[2] Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[3]
Patisiran was developed and is marketed by Alnylam.[4] The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.[5]
As of 2020, there were 1050 patients globally receiving Onpattro, generating $65.5M in net-revenues for Alnylam Pharmaceuticals.[6][7]
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.[8][9] It was approved for medical use in the United States and in the European Union in August 2018.[10][11] The per-patient cost is between US$451,430 and US$677,145 per year, depending on the number of vials needed.[12][13]
References
- ^ a b Loftus P (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. Retrieved 10 August 2018.
- ^ Kristen, Arnt V; Ajroud-Driss, Senda; Conceição, Isabel; Gorevic, Peter; Kyriakides, Theodoros; Obici, Laura (2018-11-27). "Patisiran, an RNAi therapeutic for the treatment of hereditary transthyretin-mediated amyloidosis". Neurodegenerative Disease Management. 9 (1): 5–23. doi:10.2217/nmt-2018-0033. ISSN 1758-2024. PMID 30480471.
- ^ "Onpattro (patisiran)". www.centerwatch.com. Retrieved 2021-06-18.
- ^ Reidy, Chris (October 22, 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Retrieved 5 May 2021.
- ^ New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Retrieved 16 September 2020.
- ^ "Patisiran and Vutrisiran, in development for the Treatment of Transthyretin-Mediated Amyloidosis" (PDF). Alnylam Pharmaceuticals.
{{cite web}}: CS1 maint: url-status (link) - ^ "Alnylam Pharmaceuticals Reports Fourth Quarter and Full Year 2020 Financial Results and Highlights Recent Period Activity". www.businesswire.com. 2021-02-11. Retrieved 2021-06-23.
- ^ "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.
- ^ Brooks M (10 August 2018). "FDA OKs Patisiran (Onpattro) for Polyneuropathy in hAATR". Medscape. WebMD. Retrieved 10 August 2018.
- ^ "Drug Approval Package: Onpattro (patisiran)". U.S. Food and Drug Administration (FDA). 7 September 2018. Retrieved 2 September 2020.
- ^ "Onpattro EPAR". European Medicines Agency (EMA). Retrieved 2 September 2020.
- ^ Information, National Center for Biotechnology; Pike, U. S. National Library of Medicine 8600 Rockville; MD, Bethesda (2019-08-01). Executive Summary. Canadian Agency for Drugs and Technologies in Health.
{{cite book}}: CS1 maint: numeric names: authors list (link) - ^ Lipschultz B, Cortez M (10 August 2018). "Rare-Disease Treatment From Alnylam to Cost $450,000 a Year". Bloomberg. Retrieved 11 August 2018.
External links
- "Patisiran". Drug Information Portal. U.S. National Library of Medicine.
