Patisiran is under clinical development by Alnylam Pharmaceuticals and currently in Pre-Registration for Familial Amyloid Cardiomyopathy. According to GlobalData, Pre-Registration drugs for Familial Amyloid Cardiomyopathy have a 100% phase transition success rate (PTSR) indication benchmark for progressing into Marketed. GlobalData’s report assesses how Patisiran’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Patisiran overview

Patisiran (Onpattro) is a oligonucleotide. It is formulated as injectable lipid complex solution and concentrate solution for intravenous route of administration. Onpattro is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis, in adults.

Patisiran (SAR438037, ALN-TTR02) is under development for the treatment of transthyretin amyloidosis (ATTR) with cardiomyopathy. It is administered as an intravenous infusion. ALN-TTR02 is comprised of siRNA formulated in a second-generation lipid nanoparticle (LNP). The drug candidate is an RNAi therapeutic agent targeting transthyretin (TTR). It is based on the RNAi therapeutic platform and lipid nanoparticle (LNP) platform.

Alnylam Pharmaceuticals overview

Alnylam Pharmaceuticals (Alnylam) is a biopharmaceutical company, which discovers, develops and commercializes drugs based on RNAi interference. The company’s marketed RNAi therapies include Onpattro, which is used in the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis; Givlaari for acute hepatic porphyria, and Oxlumo for primary hyperoxaluria type 1 (PH1). The company develops a broad pipeline of investigational RNAi drugs in the areas of genetic medicines, hepatic infectious, cardio-metabolic, and central nervous system (CNS) diseases. Its late-stage product candidates include vutrisiran for ATTR amyloidosis, inclisiran for hypercholesterolemia, fitusiran for hemophilia and rare bleeding disorders, and lumasiran for severe PH1. The company operates subsidiaries in North America, Europe and Asia-Pacific. Alnylam is headquartered in Cambridge, Massachusetts, the US.

For a complete picture of Patisiran’s drug-specific PTSR and LoA scores, buy the report here.


GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.