Vutrisiran is under clinical development by Alnylam Pharmaceuticals and currently in the Phase III in clinical pathway. The characteristics of the clinical trial as well as other attributes related to the drug, regulations, and company play a fundamental role in ensuring the likelihood of transition that the drug moves from its current development stage to next.
According to GlobalData, the latest event to affect Vutrisiran’s likelihood of approval (LoA) and phase transition for Familial Amyloid Cardiomyopathy took place on 22 Jun 2022, which increased the likelihood that the drug progresses to the next phase in its clinical pathway and increased the likelihood of final approval for this indication.
GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Vutrisiran Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.
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.
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