Dirloctocogene samoparvovec is under clinical development by Spark Therapeutics and currently in Phase III for Hemophilia A (Factor VIII Deficiency). According to GlobalData, Phase III drugs for Hemophilia A (Factor VIII Deficiency) have a 78% phase transition success rate (PTSR) indication benchmark for progressing into Pre-Registration. GlobalData’s report assesses how Dirloctocogene samoparvovec’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.

Dirloctocogene samoparvovec overview

RG-6357 (SPK-8011, SPK-FVIII) is under development for the treatment of hemophilia A. It is administered through intravenous route. The therapeutic candidate constitues of Adeno-associated virus (AAV) encoding coagulation factor VIII (Factor VIII). The drug candidate is a new molecular entity.

Spark Therapeutics overview

Spark Therapeutics (Spark), a subsidiary of F. Hoffmann-La Roche Ltd, is a clinical-stage gene therapy company. It provides gene therapies for inherited retinal diseases (IRDs), hemophilia and pompe disease. The company’s gene therapy vectors used in its programs are engineered using adeno-associated virus. Spark’s SPK-RPE65 is intended to cure rare blinding conditions caused by mutations in the RPE65 gene. The company provides various services such as discovery research, manufacturing, product development and regulatory advancement and U.S. commercialization. It collaborates with pharma and biotech companies for its research and development activities. The company’s gene therapy resources include Genes in Life, Genetic Alliance, Mayo Clinic, MedlinePlus, Your Genome. Spark is headquartered in Philadelphia, Pennsylvania, the US.

For a complete picture of Dirloctocogene samoparvovec’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.