How much does defibrotide sodium cost?
The cost of defibrotide sodium, sold under the brand name Defitelio, varies depending on factors such as dosage, duration of treatment, and insurance coverage. It is administered intravenously, and treatment can be lengthy, contributing to significant overall costs. For example, one report indicated that a full course of treatment for a pediatric patient with sinusoidal obstruction syndrome could range from $200,000 to $400,000 [1]. Another source suggests daily costs can be in the thousands of dollars [2].
What is defibrotide sodium used for?
Defibrotide sodium is a medication used to treat adult and pediatric patients who have sinusoidal obstruction syndrome (SOS), also known as veno-occlusive disease (VOD), with hepatic impairment following a hematopoietic stem cell transplant (HSCT) [1][3]. SOS/VOD is a serious complication that can occur after HSCT, where the small veins in the liver become blocked, leading to liver damage [3].
How does defibrotide sodium work?
The precise mechanism of action for defibrotide sodium is not fully understood [4]. However, research suggests it has anticoagulant, anti-ischemic, and thrombolytic properties [4]. It is thought to work by restoring the balance between procoagulant and anticoagulant factors on the surface of endothelial cells, which line blood vessels [4]. This may help prevent blood clots and improve blood flow in the liver [4].
What is the recommended dosage for defibrotide sodium?
The recommended dosage for defibrotide sodium is typically 6.25 mg/kg administered every six hours [3]. The dose is calculated based on the patient's weight [3]. Treatment duration is usually for a minimum of 21 days or until SOS/VOD has resolved [3].
Are there alternatives to defibrotide sodium for SOS/VOD treatment?
Historically, treatment for SOS/VOD relied on supportive care measures like fluid management and management of organ dysfunction [5]. However, defibrotide sodium is the only FDA-approved therapy specifically indicated for SOS/VOD in patients who have undergone HSCT [3][5]. Other agents have been investigated, but none have demonstrated the same level of efficacy and approval for this specific indication [5].
What are the side effects of defibrotide sodium?
Common side effects associated with defibrotide sodium treatment include low blood pressure (hypotension), diarrhea, vomiting, nausea, and fever [3]. More serious potential side effects include bleeding and hypersensitivity reactions [3].
Who manufactures defibrotide sodium?
Defibrotide sodium is manufactured by Gentium S.p.A., a subsidiary of Jazz Pharmaceuticals [3].
What is the patent status of defibrotide sodium?
Information regarding specific patent expiry dates for defibrotide sodium is not readily available in the provided context. However, pharmaceutical patents typically last for 20 years from the filing date, though extensions can be granted [6]. The development and commercialization of drugs involve complex patent landscapes that can be subject to challenges and litigation [7].
What is the success rate of defibrotide sodium in treating SOS/VOD?
Clinical studies have shown that defibrotide sodium can improve survival rates in patients with SOS/VOD following HSCT. In one pivotal study, survival at 100 days post-HSCT was significantly higher in patients treated with defibrotide sodium compared to historical controls [1][3].
Sources:
[1] https://www.ajmc.com/view/defibrotide-sodium-approved-for-rare-pediatric-condition
[2] https://www.beckershospitalreview.com/pharmacy/defitelio-price-for-sop-is-1-200-per-vial.html
[3] https://www.accessdata.fda.gov/drugsatfda_docs/label/2016/207516s000lbl.pdf
[4] https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4536176/
[5] https://pubmed.ncbi.nlm.nih.gov/26777895/
[6] https://www.uspto.gov/learning-and-resources/glossary-terms/patent-term-adjustment
[7] https://www.fiercepharma.com/legal/drug-patent-litigation-explained