See the DrugPatentWatch profile for Defibrotide
Defibrotide sodium, sold under the brand name Defitelio, is a prescription medication used to treat sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (HVOD), in hematopoietic stem cell transplant recipients [1].
How much does Defitelio cost?
The price of Defitelio can vary. A typical retail price for a vial might be around $1,000-$2,000 [2]. However, actual out-of-pocket costs for patients can be significantly lower due to insurance coverage, patient assistance programs, and pharmacy discounts [3][4]. The total cost of treatment is also influenced by the duration and dosage required for each patient [3].
What factors affect the price of Defitelio?
Several factors contribute to the price of Defitelio. As a specialized medication for a rare and severe condition, its development and manufacturing involve significant research and clinical trial costs, which are often reflected in the drug's price [5]. The price also accounts for the complexity of its production and the specialized nature of its therapeutic use [5].
Are there patient assistance programs for Defitelio?
Yes, programs are available to help eligible patients with the cost of Defitelio. Manufacturers often provide co-pay assistance programs, and there may be non-profit organizations that offer financial support for those who qualify based on income and insurance status [4]. These programs aim to make the medication more accessible to individuals facing financial challenges [4].
How is Defitelio administered?
Defitelio is administered intravenously [1]. This means it is given through an IV infusion, typically in a hospital or clinical setting [1].
What is sinusoidal obstruction syndrome (SOS)?
Sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (HVOD), is a serious and potentially life-threatening complication that can occur after a stem cell transplant [6]. It is characterized by the blockage of small veins in the liver, leading to liver damage and dysfunction [6].
Who makes Defitelio?
Defitelio is manufactured by Jazz Pharmaceuticals [7].
What are the clinical indications for Defitelio?
Defitelio is indicated for the treatment of severe sinusoidal obstruction syndrome (SOS), also known as hepatic veno-occlusive disease (HVOD), with liver dysfunction or kidney issues, following a hematopoietic stem cell transplant (HSCT) [1].
What is the mechanism of action of Defitelio?
Defitelio is a broad-spectrum deoxyribonucleic acid (DNA) derivative that is thought to work by restoring the balance of fibrinolytic and anti-thrombotic factors, thereby improving microvascular blood flow and reducing liver damage [1].
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Sources:
[1] https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/206241lbl.pdf
[2] https://www.goodrx.com/defitelio
[3] https://www.pharmacytimes.com/view/jazz-pharmaceuticals-launches-defitelio-for-sos-treatment
[4] https://www.jazzpharma.com/patients-and-caregivers/patient-support
[5] https://www.nejm.org/doi/full/10.1056/NEJMp1613361
[6] https://www.cancer.gov/publications/dictionaries/cancer-drug/defibrotide-sodium
[7] https://www.jazzpharma.com/