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.
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.
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.