Peboctocogene camaparvovec is under clinical development by Ultragenyx Pharmaceutical and currently in the Phase II 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 Peboctocogene camaparvovec’s likelihood of approval (LoA) and phase transition for Hemophilia A (Factor VIII Deficiency) took place on 14 Nov 2022, which decreased the likelihood that the drug progresses to the next phase in its clinical pathway and decreased the likelihood of final approval for this indication.

GlobalData uses proprietary data and analytics to provide a complete picture of this assessment in their Peboctocogene camaparvovec Likelihood of Approval (LoA) and Phase Transition Success Rate (PTSR) Report.

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.

Quick View Peboctocogene camaparvovec LOA Data

Report Segments
  • Innovator
Drug Name
  • Peboctocogene camaparvovec
Administration Pathway
  • Intravenous
Therapeutic Areas
  • Hematological Disorders
Key Developers
Highest Development Stage
  • Phase II

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 its clinical pathway (PTSR), as well as how likely the drug will be approved (LoA). This is based on a proprietary algorithm built from the drugs’ sales forecast, regulatory milestones, cost forecasts, WACC rate and other proprietary data sources found on GlobalData’s Pharmaceutical Intelligence Center.