Peboctocogene camaparvovec is under clinical development by Ultragenyx Pharmaceutical and currently in Phase II for Hemophilia A (Factor VIII Deficiency). According to GlobalData, Phase II drugs for Hemophilia A (Factor VIII Deficiency) have a 76% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Peboctocogene camaparvovec’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.
Peboctocogene camaparvovec overview
Peboctocogene camaparvovec (BAY-2599023) is under development for hemophilia A. It is administered intravenously as a solution concentrate. The drug candidate acts by targeting coagulation Factor VIII, in a durable fashion. It is developed based on NAV vector technology which uses adeno-associated virus hu37 (AAVhu37).
Ultragenyx Pharmaceutical overview
Ultragenyx Pharmaceutical (Ultragenyx) develops novel medicines for rare and ultra-rare genetic diseases. It has three 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). 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 Peboctocogene camaparvovec’s drug-specific PTSR and LoA scores, buy the report here.