FDA BRIEF: Week of Mar 28, 20116

FDA approved


DEFITELIO (defibrotide sodium) injection

Jazz Pharmaceuticals, Palo Alto, California, USA.

INDICATION: Treatment of adult and pediatric patients with hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following hematopoietic stem-cell transplantation (HSCT)


  • Hepatic VOD can occur in patients who receive chemotherapy and HSCT
  • Swelling and decrease in liver blood flow, liver damage
  • In most severe form, failure of kidneys, lungs
  • 80% severe hepatic VOD patients do not survive
  • Need in transplantation community to treat this rare fatal complication

REG PATHWAY: NDA, Priority Review, Orphan Drug Designation

MECHANISM OF ACTION: Not fully elucidated. Enhances the enzymatic activity of plasmin to hydrolyze fibrin, clots. Protect from damage caused by chemotherapy, tumor necrosis factor-α, serum starvation, and perfusion


  • Three studies: 2 prospective clinical trials (n=102, 75) 1 expanded access (n=351)
  • Survival at Day + 100 after HSCT : 38%,  44%, 45%  (vs. published reports of 21%-31% with no drug)


  • Serious potential side effects: Bleeding (hemorrhage) and allergic reactions
  • Should not be used in patients with bleeding complications, taking blood thinners
  • Common side effects: Abnormally low blood pressure (hypotension), diarrhea, vomiting, nausea and nosebleeds (epistaxis)











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