Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated amyloidosis,[7] a fatal rare disease that is estimated to affect 50,000 people worldwide.[8][9]
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.[8] It is a gene silencing drug that interferes with the production of an abnormal form of transthyretin.[10] Patisiran utilizes a novel approach to target and reduce production of the TTR protein in the liver via the RNAi pathway.[11]
Patisiran was developed and is marketed by Alnylam.[12] The FDA considers it to be a first-in-class medication.[13]
^ ab"Onpattro". Therapeutic Goods Administration (TGA). 2 December 2022. Archived from the original on 5 February 2023. Retrieved 28 April 2023.
^"Updates to the Prescribing Medicines in Pregnancy database". Therapeutic Goods Administration (TGA). 21 December 2022. Archived from the original on 3 April 2022. Retrieved 2 January 2023.
^"Onpattro patisiran 10 mg/5 mL concentrated injection for infusion vial (380813)". Therapeutic Goods Administration (TGA). 22 November 2022. Archived from the original on 18 December 2022. Retrieved 28 April 2023.
^"Onpattro (Alnylam Australia Pty Ltd)". Therapeutic Goods Administration (TGA). 5 December 2022. Archived from the original on 27 March 2023. Retrieved 28 April 2023.
^"Onpattro PI" (PDF). Archived from the original (PDF) on 30 May 2022.
^"Summary Basis of Decision (SBD) for Onpattro". Health Canada. 23 October 2014. Archived from the original on 31 May 2022. Retrieved 29 May 2022.
^ ab"Onpattro- patisiran injection, lipid complex". DailyMed. U.S. National Library of Medicine. 10 May 2021. Archived from the original on 4 May 2022. Retrieved 9 June 2022.
^ abLoftus P (10 August 2018). "New Kind of Drug, Silencing Genes, Gets FDA Approval". The Wall Street Journal. Archived from the original on 10 August 2018. Retrieved 10 August 2018.
^Elafros MA, Kvalsund MP, Callaghan BC (June 2022). "The Global Burden of Polyneuropathy-In Need of an Accurate Assessment". JAMA Neurology. 79 (6): 537–538. doi:10.1001/jamaneurol.2022.0565. PMC 9197927. PMID 35404377. S2CID 248083689.
^Kristen AV, Ajroud-Driss S, Conceição I, Gorevic P, Kyriakides T, Obici L (February 2019). "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. PMID 30480471.
^"Onpattro (patisiran)". www.centerwatch.com. Archived from the original on 24 June 2021. Retrieved 18 June 2021.
^Reidy C (22 October 2012). "Alnylam, Genzyme Form Alliance". The Boston Globe. Archived from the original on 7 May 2021. Retrieved 5 May 2021.
^New Drug Therapy Approvals 2018 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2019. Archived from the original on 17 September 2020. Retrieved 16 September 2020.
Patisiran, sold under the brand name Onpattro, is a medication used for the treatment of polyneuropathy in people with hereditary transthyretin-mediated...
In late 2016, the company's lead candidate in phase III studies was patisiran, a treatment targeting transthyretin (TTR) for the treatment of TTR-mediated...
European Union and in the USA. Patisiran functions similarly to inotersen. Moderate-certainty evidence suggests that patisiran mitigates worsening of peripheral...
vutrisiran to patisiran. 164 ATTRv amyloidosis patients were randomized 3:1 to subcutaneous vutrisiran 25 mg every 3 months or patisiran 0.3 mg/kg IV infusion...
developed by Ionis Pharmaceuticals and licensed to Akcea Therapeutics. Patisiran (sold under Onpattro) was developed by Alnylam Pharmaceuticals, and also...
phospholipase 2G6-associated neurodegeneration (PLAN). The FDA granted Patisiran (Onpattro) orphan drug status and breakthrough therapy designation due...
first company to have a siRNA therapy approved by the FDA. Onpattro (patisiran) was approved for the treatment of polyneuropathy of hereditary transthyretin-mediated...
disorders. Patisiran is the first double stranded siRNA-based medication approved in 2018 and developed by Alnylam Pharmaceuticals. Patisiran uses the RNAi...
and medications that decreases the activity of the transthyretin genes (patisiran and inotersen). In patients with familial transthyretin mutations, liver...
number NCT01960348 for "APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis"...
siRNA drugs. In 2018, the FDA approved Alnylam's siRNA drug Onpattro (patisiran), the first drug to use LNPs as the drug delivery system. By that point...
transthyretin familial amyloid polyneuropathy. In August 2018, the FDA approved patisiran, an siRNA-based treatment, at an expected cost of up to $450,000 per year...
million in Alnylam. Under the deal, Genzyme obtained further rights to patisiran, an RNAi treatment for transthyretin-mediated amyloidosis – a condition...
many gene therapy based drugs became available, such as Zolgensma and Patisiran. Most of these approaches utilize viral vectors, such as adeno-associated...
agreement with lower milestones and royalties, which covers Alnylam's product patisiran. Tekmira caught the world's attention and its stock rose dramatically...
compensate for the RPE65 mutation (US approval granted 2017). siRNA medicines: Patisiran (Onpattro) for the treatment of polyneuropathy in people with hereditary...
years, but it wasn’t until 2018 that the EMA approved the drug called Patisiran/Onpattro. The drug uses ds-siRNA as a mechanism of action and is deemed...
Global Information