Drug approval body Scottish Medicines Consortium (SMC) has authorised Defitelio (defibrotide) for use as a supportive treatment for haematopoietic stem cell transplantation (HSCT) in treating patients suffering from severe hepatic veno-occulusive disease (VOD).
Developed by US-based Jazz Pharmaceuticals, the Defitelio is now the only product that can be used within NHS Scotland for treating patients with VOD undergoing HSCT therapy.
SMC approval covers all diseases, including myeloma, which requires treatment with either allogeneic or autologous stem cell transplantation in Scotland.
Hepatic veno-occlusive disease is a potentially serious and complicated HSCT therapy. In severe form, it causes multi-organ failure and is life-threatening.
Every year, an estimated 3,600 patients undergo HSCT therapy in the UK, with 80% of cases being fatal.
Myeloma UK, a UK-based organisation focused on myeloma, CEO Eric Low 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," Low said.
"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."
Following SMC approval, the Scottish Health Boards have three months to decide whether or not to accept Defitelio as an approved treatment.
In addition to Defitelio (defibrotide), Jazz Pharmaceuticals also markets Erwinase drug outside the US.