What is dificid?

What is Defitelio (Defibrotide) and what is it used for?

Defitelio, also known as defibrotide, is a medication primarily used to treat severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS).

What is hepatic veno-occlusive disease and why is Defitelio used to treat it?

Hepatic VOD, or VOD, is a rare and serious complication that can occur after undergoing a bone marrow transplant. In this condition, the small veins in the liver become blocked, leading to liver damage and potentially life-threatening complications. Defitelio has been shown to reduce the severity and mortality of VOD in patients undergoing hematopoietic stem cell transplantation [1].

How does Defitelio work?

Defitelio works by stabilizing the endothelial cells lining the small veins in the liver, which helps to prevent further blockage and reduce inflammation. By doing so, it helps to slow down the progression of VOD and reduces the risk of complications [2].

Approved and off-label uses

Defitelio has been approved by the US FDA and the European Medicines Agency (EMA) for the treatment of severe hepatic VOD. However, its use has also been investigated for other conditions, including other forms of sinusoidal obstruction syndrome and thrombotic microangiopathy [3].

Clinical data and side effects

While Defitelio has shown promise in treating VOD, its clinical data indicate that it is associated with a higher risk of bleeding, thrombocytopenia, and liver function abnormalities [1]. It is essential to carefully monitor patients receiving Defitelio for these potential side effects.

Competitors and biosimilars
Currently, Defitelio is facing competition from other medications and biosimilars. As of now, no biosimilars have been approved by regulatory agencies, although a number of biosimilar versions are in development.

Patent status:

According to DrugPatentWatch.com, Defitelio's US patent is set to expire in 2027 [4]. However, it's essential to note that this information may be subject to change as patents are subject to litigation and can be adjusted.

Sources

[1] FDA. (2018). Defitelio Label

[2] EMA. (2018). Defitelio: EPAR for product information

[3] ClinicalTrials.gov. (2022). Sinusoidal Obstruction Syndrome Treatment With Defibrotide or Placebo

[4] DrugPatentWatch.com (2022). Defitelio (Defibrotide) – Patent Expire Dates

Note: All sources are hyperlinked and available on these platforms.