Dublin, Ireland, June 9, 2014,
Jazz Pharmaceuticals plc today announced that the Scottish Medicines Consortium (SMC) has accepted Defitelio®(defibrotide) for use within NHS Scotland1
Defibrotide is the first and only 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 (HSCT) therapy.2 (NB. HSCT therapy is now the preferred clinical term used for bone marrow transplantation)
Defibrotide has been accepted with no restrictions – and in line with its indication – following its first submission to the SMC1,2
Hepatic veno-occlusive disease is a potentially serious, early complication of HSCT therapy 3,4,5 In its severe form, VOD is life-threatening and is associated with multi-organ failure3,4,5 Severe VOD can be fatal in over 80% of patients4,5 Approximately 3,600 patients undergo HSCT therapy in the UK every year6
Commenting on the SMC decision, Eric Low, chief executive, Myeloma UK (based in Edinburgh) 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.”
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions.
What is veno-occlusive disease (VOD)?
Hepatic veno-occlusive disease (VOD) – also known as sinusoidal obstruction syndrome (SOS) – is a potentially serious, early complication in patients undergoing hematopoietic stem cell transplantation (HSCT). In the EU, VOD is designated as a rare disease, affecting less than five in 10,000 people. Studies have reported a wide range of incidence rates for VOD. Generally, data indicates that up to 14% of patients undergoing HSCT
go on to develop VOD.
Severe VOD In its severe form (sVOD), the disease is life-threatening and is associated with multi-organ failure and death in over 80% of patients.
About Defitelio® (defibrotide)
Defitelio® (defibrotide) is the first and only licensed product for the treatment of severe hepatic venoocclusive disease (sVOD) in haematopoietic stem cell transplantation (HSCT). It is indicated for adults and
children over one month of age.
HSCTs are performed with curative intent in patients with haematological malignancies, selected solid tumours and some non-malignant disorders such as serious haemaglobinopathies. HSCT can have a wide variety of complications. Hepatic veno-occlusive disease (VOD) is an early complication in patients undergoing HSCT. In its severe form, VOD is life-threatening and is associated with multi-organ failure and death in over 80% of patients. In the EU, VOD is designated as a rare disease, affecting less than five in 10,000 people. Studies have reported a wide range of incidence rates for VOD. Generally, data indicates that up to 14% of patients undergoing HSCT may go on to develop VOD.
Clinical Study Results
In the pivotal clinical trials for Defitelio®, it was shown to provide a significant increase in survival rates for patients with sVOD in HSCT. The results demonstrated a 52% increase in survival at 100 days after transplantation for patients treated with Defitelio compared to patients in the historical control group (38.2% in the Defitelio group vs. 25.0% in the historical control group; p= 0.0341). In the clinical trial, 23.5% of patients treated with Defitelio achieved complete response at 100 days after transplantation versus 9.4% of patients in the historical control group (p=0.013). The treatment has generally been well tolerated in all age groups. In the Phase 3 pivotal treatment study, the overall incidence of adverse events was similar in the Defitelio treatment group and in the control group. The most frequent adverse events observed during clinical trials were haemorrhage, hypotension and coagulopathy. Please consult the SmPC for the full list of all side effects reported with Defitelio.
Defitelio European Approval
In October 2013, the European Commission granted Marketing Authorisation under exceptional circumstances for Defitelio® (defibrotide) for the treatment of severe hepatic veno-occlusive disease in haematopoietic stemcell transplantation therapy. It is indicated in patients over one month of age. Defitelio is not indicated in patients with hypersensitivity to defibrotide or any of its excipients or with concomitant use of thrombolytic
About Jazz Pharmaceuticals plc
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a specialty biopharmaceutical company focused on improving patients’ lives by identifying, developing and commercializing differentiated products that address unmet medical needs. The company has a diverse portfolio of products and/or product candidates in the areas of sleep, haematology/oncology, pain and psychiatry. The company’s U.S. marketed products in these areas include: Xyrem® (sodium oxybate) oral solution, Erwinaze® (asparaginase Erwinia chrysanthemi), Prialt® (ziconotide) intrathecal infusion, Versacloz™ (clozapine) oral suspension, FazaClo® (clozapine, USP) HD and FazaClo LD. Jazz Pharmaceuticals also has a number of products marketed outside the United States, including Erwinase® and Defitelio® (defibrotide).
For more information, please visit www.jazzpharmaceuticals.com.
- Scottish Medicines Consortium. Detailed Advice Document. www.scottishmedicines.org.uk (as at 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;16:157–168
- Richardson PG et al. Ther Adv Hematol. 2012 3 (4) 253-265
- British Society of Bone Marrow Transplantation. Accessed at: http://bsbmt.org/2012-activity/