FDA approves first treatment for rare disease in patients who receive stem cell transplant from blood or bone marrow
March 30, 2016 - 1:20 PM
WASHINGTON, D.C. - The U.S. Food and Drug Administration today approved Defitelio (defibrotide sodium) to treat adults and children who develop hepatic
veno-occlusive disease (VOD) with additional kidney or lung
abnormalities after they receive a stem cell transplant from blood or
bone marrow called hematopoietic stem cell transplantation (HSCT). This is the first FDA-approved therapy for treatment of severe hepatic VOD, a rare and life-threatening liver condition.
is a procedure performed in some patients to treat certain blood or
bone marrow cancers. Immediately before an HSCT procedure, a patient
receives chemotherapy. Hepatic VOD can occur in patients who receive
chemotherapy and HSCT. Hepatic VOD is a condition in which some of the
veins in the liver become blocked, causing swelling and a decrease in
blood flow inside the liver, which may lead to liver damage. In the most
severe form of hepatic VOD, the patient may also develop failure of the
kidneys and lungs. Fewer than 2 percent of patients develop severe
hepatic VOD after HSCT, but as many as 80 percent of patients who
develop severe hepatic VOD do not survive.
approval of Defitelio fills a significant need in the transplantation
community to treat this rare but frequently fatal complication in
patients who receive chemotherapy and HSCT,” said Richard Pazdur, M.D.,
director of the Office of Hematology and Oncology Products in the FDA’s
Center for Drug Evaluation and Research.
The efficacy of
Defitelio was investigated in 528 patients treated in three studies: two
prospective clinical trials and an expanded access study. The patients
enrolled in all three studies had a diagnosis of hepatic VOD with liver
or kidney abnormalities after HSCT. The studies measured the percentage
of patients who were still alive 100 days after HSCT (overall survival).
In the three studies, 38 to 45 percent of patients treated with
Defitelio were alive 100 days after HSCT. Based on
published reports and analyses of patient-level data, the expected
survival rates 100 days after HSCT would be 21 to 31 percent for
patients with severe hepatic VOD who received only supportive care or
interventions other than Defitelio.
The most common side effects
of Defitelio include abnormally low blood pressure (hypotension),
diarrhea, vomiting, nausea and nosebleeds (epistaxis). Serious potential
side effects of Defitelio that were identified include bleeding
(hemorrhage) and allergic reactions. Defitelio should not be used in
patients who are having bleeding complications or who are taking blood
thinners or other medicines that reduce the body’s ability to form
The FDA granted the Defitelio application priority review status,
which facilitates and expedites the development and review of certain
drugs in light of their potential to benefit patients with serious or
life-threatening conditions. Defitelio also received orphan drug designation,
which provides incentives such as tax credits, user fee waivers and
eligibility for exclusivity to assist and encourage the development of
drugs for rare diseases.
Defitelio is marketed by Jazz Pharmaceuticals based in Palo Alto, California.
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