U.S. FDA approves first drug for a rare kind of liver disorder

• United States Food and Drug Administration approves a drug for a rare type of liver disorder


• Jazz Pharmaceuticals Plc’s defibrotide targets patients with hepatic veno-occlusive disease 


• It is the first drug that got the US approval for the condition

The United States Food and Drug Administration (U.S. FDA) has approved a drug for a rare type of liver disorder; making it the first treatment for the illness.

“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,” U.S. FDA said in a news release.

In a Reuters article which was posted on GMA News, it was disclosed that U.S FDA approved Jazz Pharmaceuticals Plc’s defibrotide to treat patients with hepatic VOD which is a potentially fatal complication of stem-cell transplantation in which some of the veins in the liver become blocked, causing swelling and a decrease in blood flow inside the liver.

The disease is not something to be ignored as it can lead to liver damage. In addition, in its most severe form, patients develop failure of the kidneys and lungs.

According to Stifel Nicolaus analyst Annabel Samimy while citing a published research, out of the nearly 20,000 stem cell transplantation procedures done in the United States annually, about 8.8 to 13.8 percent of the patients suffer from severe VOD. With this, she said she expects the drug to be launched in the United States real soon.

Richard Pazdur, M.D., director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research said 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.

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