Defitelio Approved for Hepatic Veno-Occlusive Disease
March 30, 2016 – The U.S. Food and Drug Administration (FDA) approved Defitelio® (defibrotide sodium) injection for intravenous (IV) use. Manufactured by Jazz Pharmaceuticals, Defitelio is approved under priority review and with orphan-drug status indicated to treat hepatic veno-occlusive disease (VOD) for adults and children with renal or pulmonary dysfunction following a hematopoietic stem-cell transplant (HSCT). Defitelio is the first drug that is FDA approved to treat severe hepatic VOD.
Defitelio is available in 200mg/2.5mL (80mg/mL) single-use vials and the recommended dose is 6.25mg/kg administered as a two-hour intravenous infusion once every six hours for at least 21 days until symptoms of VOD are resolved or up to a maximum of 60 days. With VOD, also known as sinusoidal obstruction syndrome, veins in the liver become blocked, restricting blood flow and causing the liver to swell, which may lead to liver damage. Kidney and lung function can become impaired as well. In the United States, approximately 20,000 patients undergo hematopoietic stem-cell transplant (HSCT) each year. Between 9% and 14% of patients experience serious VOD, which can be fatal. Because Defitelio may cause hemorrhaging, it should not be used in patients being treated with anticoagulants or fibrinolytic therapies.
Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by regular mail or by fax.
- Complete and submit the report Online: www.fda.gov/medwatch/report.htm
- Regular Mail or Fax: Download form www.fda.gov/MedWatch/getforms.htm or call 1-800-332-1088 to request a reporting form, then complete and return to the address on the pre-addressed form, or submit by fax to 1-800-332-0178
Actual drug patent expiration dates and a availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval. Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval. Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability.
*This is provided for information only. The reference to any medication above does not mean the medication is covered by your plan.