Silver Spring, MD (Press Release) – 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.

HSCT 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.

“The 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 clots.

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.

The FDA, an agency within the U.S. Department of Health and Human Services, promotes and protects the public health by, among other things, assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological prod­ucts for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supple­ments, prod­ucts that give off electronic radiation, and for regulating tobacco prod­ucts.

Source: Food and Drug Admin­istra­tion.