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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)

DUBLIN, March 30, 2016 /PRNewswire/ -- Jazz Pharmaceuticals plc (JAZZ) today announced that the United States (U.S.) Food and Drug Administration (FDA) granted marketing approval for Defitelio® (defibrotide sodium) for the treatment of adult and pediatric patients with hepatic VOD, also known as sinusoidal obstruction syndrome (SOS), with renal or pulmonary dysfunction following HSCT.1

"FDA's approval of Defitelio underscores the importance of Defitelio to children and adults as the first and only proven treatment for this rare and often deadly complication of stem-cell transplantation. Defitelio is a clinically significant therapeutic advance because it is a potentially curative intervention for patients with VOD, which may save lives with a single course of therapy. Before today, patients in the U.S. had no approved options," said Bruce Cozadd, chairman and chief executive officer of Jazz Pharmaceuticals. "The commercial availability of Defitelio in the U.S. demonstrates Jazz Pharmaceuticals' continued commitment to bringing meaningful new treatments to patients to fill high unmet medical needs."

The FDA approval of Defitelio is supported by efficacy data from three clinical studies in patients with hepatic VOD with renal or pulmonary dysfunction following HSCT who were treated with Defitelio at the recommended 6.25 mg/kg every 6 hours; results are provided in the table below.1


Study Design

Patient
Number

Survival Rate at
Day +100 after
HSCT

Confidence Interval (CI)

Study 1

Phase 3 Prospective Study

n=102

38%

95% CI: 29%, 48%

Study 2

Phase 2 Prospective Study

n=75

44%

95% CI: 33%, 55%

Study 3

Expanded Access Study

n=351

45%

95% CI: 40%, 51%

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 Paul G. Richardson, M.D., director of clinical research at the LeBow Institute for Myeloma Therapeutics and the Jerome Lipper Multiple Myeloma Center at the Dana-Farber Cancer Institute and the RJ Corman Professor of Medicine at Harvard Medical School. "Defitelio thus fulfills an unmet need having shown a consistent Day +100 patient survival benefit across three large prospective studies. Importantly, Defitelio provides transplant teams with the first approved treatment option that can help return patients to the road to recovery."

"We applaud the FDA's approval of Defitelio and acknowledge Jazz Pharmaceuticals' commitment to bringing this meaningful new treatment to the small number of patients who develop VOD with renal or pulmonary dysfunction following stem-cell transplantation," said Susan K. Stewart, Executive Director at BMT InfoNet (Blood & Marrow Transplant Information Network).

Shipments of Defitelio to distribution channels will commence within a week.

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