Pariglasgene brecaparvovec is under clinical development by Ultragenyx Pharmaceutical and currently in Phase III for Glycogen Storage Disease 1A. According to GlobalData, Phase III drugs for Glycogen Storage Disease 1A does not have sufficient historical data to build an indication benchmark PTSR for Phase III. GlobalData uses proprietary data and analytics to create drugs-specific PTSR and LoA in the Pariglasgene brecaparvovec LoA Report. Buy the report here.
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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.
Pariglasgene brecaparvovec overview
Pariglasgene brecaparvovec (DTX-401) is under development for the treatment of glycogen storage disease type 1a. It is administered through intravenous route as infusion. The therapeutic comprises of adeno-associated virus vector (AAV) type 8 encoding G6PC gene. It is developed based on NAV vector technology.
Ultragenyx Pharmaceutical overview
Ultragenyx Pharmaceutical (Ultragenyx) is a biotechnology company that develops novel medicines for rare and ultra-rare genetic diseases. It has four marketed products, Mepsevii (vestronidase alfa) for the treatment of Mucopolysaccharidosis (Sly syndrome); Dojolvi (triheptanoin), a highly purified, synthetic, 7-carbon fatty acid triglyceride for long-chain fatty acid oxidation disorders (LC-FAOD); Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH); and Evkeeza (evinacumab) for the treatment of homozygous familial hypercholesterolemia (HoFH). Its pipeline includes DTX401 for the treatment of glycogen storage disease type Ia, or GSDIa; UX701 for Wilson disease; UX053 for glycogen storage disease type III; and GTX-102 for Angelman syndrome in partnership with GeneTx. It works in partnership with various academic institutes and biotechnology companies to advance its product candidates. Ultragenyx is headquartered in Novato, California, the US.
For a complete picture of Pariglasgene brecaparvovec’s drug-specific PTSR and LoA scores, buy the report here.
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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.
