Jazz Pharmaceuticals has kicked off the European rollout of its rare liver disease drug Defitelio (defibrotide), with launches this week in Germany and Austria.
Defitelio is the first licensed product for the treatment of severe hepatic veno-occlusive disease (severe VOD or sVOD) in patients over one month of age undergoing haematopoietic stem cell transplantation therapy (HSCT).
Severe VOD, one of the most serious early complications in HSCT therapy (used in patients with haematological malignancies, selected solid tumours and some non-malignant disorders), is associated with multi-organ failure and is fatal in over 80% of patients.
“Severe VOD is a complex and unpredictable disease, and its impact on patients, physicians and resources is substantial," says Professor Mohamad Mohty, President-Elect of the EBMT and Professor of Haematology, Saint-Antoine Hospital and University Pierre & Marie Curie, Paris.
Clinical trials have shown that Defitelio can provide a significant increase in survival rates for patients, with data showing a 52% increase in survival at 100 days after transplantation for patients given the drug compared with those in the historical control group.
"Early and effective intervention is crucial in saving lives and limiting the potentially significant burden of this disease, and physicians have been eagerly awaiting the commercial availability of Defitelio in Europe,” Prof Mohty noted.
Jazz, which picked up ownership of the drug via its purchase of Italy's Gentium earlier this year, said it expects to continue the launch in 27 additional European countries on a rolling basis during 2014 and 2015.