Defitelio's price is not publicly disclosed by its manufacturer, Jazz Pharmaceuticals [1]. The cost can vary based on several factors, including the specific treatment regimen and pharmacy [2].
How is Defitelio administered and what is it used for?
Defitelio is an intravenous infusion used to treat sinusoidal obstruction syndrome (SOS), also known as veno-occlusive disease (VOD), in pediatric and adult patients who have undergone a hematopoietic stem cell transplant (HSCT) [3]. SOS/VOD is a serious complication that can occur after HSCT, where the small veins in the liver become blocked [3].
What are the potential side effects of Defitelio?
Common side effects of Defitelio include low blood pressure (hypotension), diarrhea, vomiting, nausea, and febrile neutropenia, which is a low white blood cell count with fever [3]. Less common but serious side effects may include bleeding [3].
What is the mechanism of action of Defitelio?
Defitelio is a deoxyribonucleic acid (DNA) containing herbal product that is thought to exert its effects by breaking down blood clots and restoring blood flow in the liver [4]. It helps to prevent the blockage of small veins in the liver, which is characteristic of SOS/VOD [3].
How is Defitelio dosed?
The recommended dose of Defitelio is 6.25 mg/kg infused intravenously over 2 hours every 6 hours [3]. Treatment is typically continued until SOS/VOD resolves or for a maximum of 60 days [3].
Where can I find patient assistance programs for Defitelio?
Jazz Pharmaceuticals, the manufacturer of Defitelio, offers a patient assistance program called Jazz Cares. This program aims to help eligible patients access the medication [1][5]. Information on eligibility and how to enroll can be found on the Jazz Pharmaceuticals website [5].
What is the typical length of Defitelio treatment?
Treatment with Defitelio is generally continued until the patient's sinusoidal obstruction syndrome (SOS)/veno-occlusive disease (VOD) resolves or for a maximum of 60 days [3].
What is the success rate of Defitelio in treating SOS/VOD?
Clinical studies have shown that Defitelio can improve survival rates in patients with severe SOS/VOD following HSCT [3][4]. One pivotal trial demonstrated a survival benefit at day 100 for patients treated with Defitelio compared to historical controls [4].
Are there alternative treatments for SOS/VOD?
While Defitelio is the only FDA-approved drug for SOS/VOD, supportive care measures are also crucial. These can include fluid management, pain control, and management of other complications arising from HSCT [3]. Other agents have been investigated but are not FDA-approved for this indication [4].
What regulatory approvals does Defitelio have?
Defitelio received FDA approval in March 2016 for the treatment of severe SOS/VOD in pediatric and adult patients following HSCT [3][4]. It has also received approval in Europe and other regions [3].
Sources:
[1] https://www.jazzpharma.com/media/press-releases/jazz-pharmaceuticals-announces-defitelio-defibrotide-approved-by-the-u-s-food-and-drug-administration-for-the-treatment-of-patients-with-severe-hepatic-veno-occlusive-disease-v-o-d-pediatric-and-adult-patients-following-hematopoietic-stem-cell-transplantation-hsct
[2] https://www.drugs.com/price-guide/defitelio
[3] https://www.defitelio.com/
[4] https://www.fda.gov/drugs/drug-approvals-and-databases/defitelio-defibrotide-solution
[5] https://www.jazzcares.com/