Vutrisiran is under clinical development by Alnylam Pharmaceuticals and currently in Phase III for Familial Amyloid Cardiomyopathy. According to GlobalData, Phase III drugs for Familial Amyloid Cardiomyopathy have a 75% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Vutrisiran’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.
Smarter leaders trust GlobalData
Vutrisiran overview
Vutrisiran (Amvuttra) is designed to target and silence specific messenger RNA, blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. It is formulated as solution for subcutaneous route of administration, Amvuttra indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Vutrisiran (ALN-TTRsc02) is under development for the treatment of familial amyloid polyneuropathy (FAP/hereditary transthyretin amyloidosis), transthyretin amyloidosis (ATTR) with cardiomyopathy and Stargardt disease. It is administered subcutaneously. It is an RNAi oligonucleotide agent targeting transthyretin (TTR) and developed based on second generation Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.
See Also:
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 Vutrisiran’s drug-specific PTSR and LoA scores, buy the report here.
Premium Insights
From
The gold standard of business intelligence.
Blending expert knowledge with cutting-edge technology, GlobalData’s unrivalled proprietary data will enable you to decode what’s happening in your market. You can make better informed decisions and gain a future-proof advantage over your competitors.
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 clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
