Dirloctocogene samoparvovec is under clinical development by Spark Therapeutics 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 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 part of F. Hoffmann-La Roche Ltd, is a clinical-stage gene therapy company. It provides gene therapies for inherited retinal diseases (IRDs), hemophilia, 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.