Here are four things to know:
1. The drug is intended for adults and children who develop severe hepatic VOD with additional kidney or lung abnormalities after they receive a stem cell transplant through a hematopoietic stem cell transplantation procedure.
2. Fewer than 2 percent of patients develop severe hepatic VOD after hematopoietic stem cell transplantation, but as many as 80 percent of patients who develop severe hepatic VOD do not survive.
3. The efficacy of Defitelio was investigated in 528 patients treated in three studies.
4. Defitelio is marketed by Jazz Pharmaceuticals based in Palo Alto, Calif.
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