Rebisufligene etisparvovec is under clinical development by Ultragenyx Pharmaceutical and currently in Phase III for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ). According to GlobalData, Phase III drugs for Mucopolysaccharidosis III (MPS III) (Sanfilippo Syndrome ) 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 Rebisufligene etisparvovec LoA Report. 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.
Rebisufligene etisparvovec overview
Rebisufligene etisparvovec (ABO-102) is under development for the treatment of Sanfilippo syndrome type A. It is administered through intravenous injection. The therapeutic candidate is a gene therapy that comprises of recombinant adeno-associated virus vector serotype 9 (AAV9) expressing human sulfoglucosamine sulfohydrolase.
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) and Crysvita (burosumab), a fully human monoclonal antibody for the treatment of X-linked hypophosphatemia (XLH); 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 Rebisufligene etisparvovec’s drug-specific PTSR and LoA scores, buy the report here.
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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.
