Jazz Pharmaceuticals has announced that the Scottish Medicines Consortium (SMC) has accepted Defitelio® (defibrotide) for use within NHS Scotland.(1)
Defibrotide is the first and only licensed product for the treatment of severe hepatic veno-occlusive disease (severe VOD or sVOD) in patients over 1 month of age undergoing haematopoietic stem cell transplantation (HSCT) therapy.(2)
Defibrotide has been accepted with no restrictions – and in line with its indication – following its first submission to the SMC.(1,2)
Hepatic veno-occlusive disease is a potentially serious, early complication of HSCT therapy.(3–5)
In its severe form, VOD is life-threatening and is associated with multi-organ failure.(3–5) Severe VOD can be fatal in over 80% of patients.(4,5)
Approximately 3,600 patients undergo HSCT therapy in the UK every year.(6)
Commenting on the SMC decision, Eric Low, chief executive, Myeloma UK, said: “The SMC’s approval of Defitelio is good news for Scottish patients undergoing HSCT therapy. For this group of people, often including children as well as adults, the prospect of undergoing such an intensive procedure can be a very uncertain and worrying time. It is therefore important that clinicians have supportive treatments available to allay fears associated with transplantation and to treat life-threatening complications of therapy, such as severe VOD, as and when they arise.”
- Scottish Medicines Consortium. Detailed Advice Document. Available at: www.scottishmedicines.org.uk (accessed 9th June 2014).
- Defitelio® Summary of Product Characteristics, 2013.
- Carreras E. Chapter 11: Early complications after HSCT. EBMT-ESH Handbook, 2012.
- Coppell JA, et al. Biol Blood Marrow Transplant 2010;16:157–68.
- Richardson PG et al. Ther Adv Hematol. 2012;3:253-65.
- British Society of Bone Marrow Transplantation. Available at: http://bsbmt.org/2012-activity/ (accessed 9th June 2014).