Defibrotide sodium is under clinical development by Jazz Pharmaceuticals and currently in Phase II for Sickle Cell Disease. According to GlobalData, Phase II drugs for Sickle Cell Disease have a 59% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Defibrotide sodium’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.

Defibrotide sodium overview

Defibrotide sodium (Noravid, Defitelio, Dinelasi, Prociclide) is a sodium salt of a mixture of single-stranded oligodeoxyribonucleotides derived from porcine mucosal DNA with antithrombotic, anti-inflammatory, anti-ischemic properties. It is formulated as concentrate for a solution and as an injection for intravenous administration and hard gelatin capsule for oral administration. Defibrotide is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstructive syndrome (SOS) in hematopoietic stem-cell transplantation (HSCT) therapy.

Defibrotide sodium is under development for the prevention of acute respiratory distress syndrome and pneumonia due to Covid 19, sickle cell disease and beta thalassemia, hepatic veno-occlusive disease in adult and pediatric patients undergoing hematopoietic stem cell transplant, mucocutaneous lymph node syndrome (Kawasaki Disease). It was also under development for the treatment of multiple myeloma, transplant-associated thrombotic microangiopathy and thrombotic thrombocytopenic purpura. It was also under development for veno-occlusive disease (VOD), unspecified indication (exploratory activities) and CAR-T-associated neurotoxicity and graft versus host disease.

Jazz Pharmaceuticals overview

Jazz Pharmaceuticals is a specialty biopharmaceutical company which identifies, develops, and commercializes medicines with focus on narcolepsy, cancer, pain, and psychiatry. Its marketed products include Xyrem (sodium oxybate) oral solution, Sunosi (solriamfetol), Erwinaze (asparaginase erwinia chrysanthemi), Defitelio (defibrotide sodium), Vyxeos (daunorubicin and cytarabine) liposome for injection, and others. The company also has various pipeline product candidates related to focus areas such as sleep, neurological disorders, hematology, and oncology including hematologic malignancies and solid tumors. The company sells its products through a network of local distributors and wholesalers. It has operations in the US and Europe. Jazz Pharmaceuticals is headquartered in Dublin, Ireland.

For a complete picture of Defibrotide sodium’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 18 March 2024

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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.