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|Jazz Pharmaceuticals Announces FDA Approval of Defitelio® (defibrotide sodium) for the Treatment of Hepatic Veno-Occlusive Disease (VOD) with Renal or Pulmonary Dysfunction Following Hematopoietic Stem-Cell Transplantation (HSCT)|
The safety of Defitelio to support approval is based on data from 176 patients in the clinical development program for the treatment of VOD with renal and/or pulmonary dysfunction following HSCT who were treated with Defitelio. The most common adverse reactions (incidence ≥10% and independent of causality) with Defitelio treatment were hypotension (low blood pressure), diarrhea, vomiting, nausea and epistaxis (nose bleeds).1 The most common serious adverse reactions (incidence ≥5% and independent of causality) were hypotension (11%) and pulmonary alveolar hemorrhage (7%).1
"VOD/SOS is a devastating condition, which can develop without warning after stem-cell transplantation and can progress rapidly causing severe kidney or lung dysfunction and lead to multi-organ failure. Thus, it can derail a patient's recovery from the curative intent of a stem-cell transplant, with patients who develop VOD/SOS and multi-organ failure facing an overall mortality rate of over 80%," said
"We applaud the
Shipments of Defitelio to distribution channels will commence within a week.
Defitelio® (defibrotide sodium) injection 80mg/mL is indicated for the treatment of adult and pediatric patients with hepatic VOD, also known as SOS, with renal or pulmonary dysfunction following HSCT.1 The FDA granted the Defitelio application priority review status. Defitelio also received orphan drug designation for the treatment of hepatic VOD.
Important Safety Information1
Defitelio should not be given to patients who are:
Defitelio may increase the risk of bleeding in patients with VOD and should not be given to patients with active bleeding. During treatment with Defitelio, patients should be monitored for signs of bleeding. In the event that bleeding occurs during treatment with Defitelio, treatment should be temporarily or permanently stopped. Patients should tell the doctor right away about any signs or symptoms of hemorrhage such as unusual bleeding, easy bruising, blood in urine or stool, headache, confusion, slurred speech, or altered vision.
Defitelio may cause allergic reactions including anaphylaxis. Patients who develop signs and symptoms of anaphylaxis such as trouble breathing, severe itching, skin rash or hives, or swelling of the face, lips, mouth or tongue should seek medical attention immediately.
The most common side effects of Defitelio are decreased blood pressure, diarrhea, vomiting, nausea and nose bleeds. Please see full Prescribing Information for Defitelio before prescribing.
▼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 via the national reporting system found under section 4.8 of the SmPC. (http://www.ema.europa.eu/ema/index.jsp?curl=/pages/medicines/human/medicines/002393/human_med_001646.jsp)
HSCT is an aggressive, potentially curative procedure to treat patients with malignant and non-cancerous hematologic disorders such as leukemia, lymphoma and aplastic anemia, and congenital immunodeficiency and autoimmune disorders.2 VOD is a rare complication of HSCT, which occurs in approximately 9–14% of HSCT patients.3,4 Hepatic VOD, also known as SOS, is an early and life-threatening complication affecting the sinusoidal endothelial cells of the liver, which can typically occur within the first 21 days following HSCT.4,5 Hepatic VOD progresses to become life-threatening in approximately 30-50% of cases.5 VOD with multi-organ dysfunction (MOD) is associated with an overall mortality (death) rate of 84%.3 MOD is characterized by the presence of renal or pulmonary dysfunction.6,7 VOD is often characterized by sudden weight gain, hepatomegaly (abnormally enlarged liver), and elevated bilirubin.6,7
"Safe Harbor" Statement under the Private Securities Litigation Reform Act of 1995
This press release contains forward-looking statements, including, but not limited to, statements related to the potential benefits and commercial availability of Defitelio in the U.S. and other statements that are not historical facts. These forward-looking statements are based on the company's current plans, objectives, estimates, expectations and intentions, and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the company's ability to effectively commercialize Defitelio in the U.S.; delays or problems in the supply or manufacture of Defitelio; obtaining and maintaining appropriate pricing and reimbursement; complying with the requirements of regulatory agencies; the challenges of achieving and maintaining commercial success of Defitelio; and other risks and uncertainties affecting the company, including those described from time to time under the caption "Risk Factors" and elsewhere in
2 Ikehara S. New strategies for BMT and organ transplantation. Int J Hematol. 2002;76(Suppl 1):161-4.
3 Coppell JA, Richardson PG, Soiffer R, et al. Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant. 2010;16(2):157-168.
4Tsirigotis PD, Resnick IB, Avni B, et al. Incidence and risk factors for moderate-to-severe veno-occlusive disease of the liver after allogeneic stem cell transplantation using a reduced intensity conditioning regimen. Bone Marrow Transplant. 2014;49(11):1389-1392.
5 Carreras E, Díaz-Beyá M, Rosiñol L, et al. The incidence of veno-occlusive disease following allogeneic hematopoietic stem cell transplantation has diminished and the outcome improved over the last decade. Biol Blood MarrowTransplant. 2011;17(11):1713-1720.
6 Carreras E. How I manage sinusoidal obstruction syndrome after haematopoietic cell transplantation. Brit J Haematol. 2015 Feb.; 168 (4); 481-91.
7 Mohty M, Malard F, Abecassis M, et al. Sinusoidal obstruction syndrome/venoocclusive disease: current situation and perspectives—a position statement from the
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