Givosiran is a Antisense RNAi Oligonucleotide owned by Alnylam Pharmaceuticals, and is involved in 8 clinical trials, of which 5 were completed, 2 are ongoing, and 1 is planned.
Givosiran targets the aminolevulinate synthase 1 (ALAS-1). It knocks down of ALAS-1 mRNA and completly inhibits the toxic intermediates that mediate the symptoms and pathology of acute intermittent porphyria (AIP). The ALAS1 gene provides instructions for making an enzyme called delta-aminolevulinate synthase 1 or ALA-synthase. The drug candidate inhibits the ALAS-1 and controls Porphobilinogen deaminase (PBGD) and lowers the accumulation of heme precursors.
The revenue for Givosiran is expected to reach a total of $8.4bn through 2038. This change impacts the valuation of this asset and is an important factor to understand the current value of the drug in a clinical process. View the complete picture with the Givosiran NPV Report.
Givosiran is originated and owned by Alnylam Pharmaceuticals.
Givosiran sodium (Givlaari) is a hepatoprotective agent. It is formulated as solution for subcutaneous route of administration. Givosiran is indicated for the treatment of adults with acute hepatic porphyria (AHP). Givlaari is indicated for the treatment of acute hepatic porphyria (AHP) in adults and adolescents aged 12 years and older.
It is under development for the treatment of hepatic porphyrias including acute intermittent porphyria (AIP), hereditary corproporhyria, variegate porphyria, Delta-aminolevulinic acid dehydratase deficient porphyria in Japan. The drug candidate is administered subcutaneously. It is a RNAi therapeutic targeting ALAS-1, a liver-expressed, rate-limiting enzyme upstream of PBGD in the heme biosynthesis pathway. It is developed based on 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.
The company reported revenues of (US Dollars) US$844.3 million for the fiscal year ended December 2021 (FY2021), an increase of 71.3% over FY2020. The operating loss of the company was US$708.7 million in FY2021, compared to an operating loss of US$828.4 million in FY2020. The net loss of the company was US$852.8 million in FY2021, compared to a net loss of US$858.3 million in FY2020. The company reported revenues of US$264.3 million for the third quarter ended September 2022, an increase of 17.6% over the previous quarter.
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