See the DrugPatentWatch profile for Defibrotide
Defibrotide's marketing exclusivity in Europe ended on January 31, 2024 [1]. This development follows the expiration of its patent protection in the region [1].
What does loss of exclusivity mean for Defitelio in Europe?
With the loss of exclusivity, Defitelio, also known by the brand name Defitelio, is now open to competition from generic or biosimilar versions in the European market [1]. This means that other pharmaceutical companies may seek to produce and market their own versions of defibrotide, potentially leading to increased availability and lower prices for the treatment [1].
What is Defitelio used for?
Defitelio is a medication used to treat adult and pediatric patients with severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), which is a complication following hematopoietic stem cell transplantation [2]. It is also used in children and adults for the treatment of severe VOD/SOS [3].
When did Defibrotide's patent protection expire in Europe?
Defibrotide's patent protection in Europe expired on January 31, 2024 [1]. This date marks the end of the period during which the innovator company held exclusive rights to market the drug [1].
Can generic versions of Defitelio be introduced in Europe now?
Yes, with the loss of marketing exclusivity and patent expiry, generic manufacturers can now apply for authorization to market their versions of defibrotide in Europe [1]. Regulatory approval processes will still need to be followed, but the path is now open for generic competition [1].
What are the implications of Defitelio losing exclusivity for patients?
The loss of exclusivity for Defitelio may lead to increased competition, which can potentially result in lower drug prices [1]. This could improve patient access to the treatment in Europe [1]. Patients may also have more treatment options as different manufacturers enter the market [1].
How does Defitelio work?
Defibrotide is a deoxyribonucleic acid-derived active substance that acts as a non-competitive inhibitor of poly(ADP-ribose) polymerase (PARP) and has anticoagulant and fibrinolytic properties [3]. It also exhibits antithrombotic, anti-inflammatory, and cytoprotective effects [3]. These actions are believed to contribute to its therapeutic benefits in treating VOD/SOS by restoring vascular integrity and promoting fibrinolysis [3].
What is Veno-occlusive Disease (VOD)/Sinusoidal Obstruction Syndrome (SOS)?
Veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), is a serious and potentially life-threatening complication that can occur after hematopoietic stem cell transplantation (HSCT) [2][3]. It is characterized by damage to the small veins (sinusoids) in the liver, leading to obstruction and congestion [2][3]. Symptoms can include jaundice, liver enlargement, and fluid accumulation in the abdomen (ascites) [2].
Who developed Defitelio?
Defitelio was developed by Jazz Pharmaceuticals [4].
What are the clinical data on Defitelio?
Clinical studies have demonstrated the efficacy of Defitelio in treating severe VOD/SOS. In a pivotal study, Defitelio showed a survival benefit in patients with severe VOD/SOS [2]. Data from post-marketing surveillance and real-world evidence also contribute to understanding its use and effectiveness [3].
What are the risks associated with Defitelio treatment?
Common side effects of Defitelio include hypotension, hypertension, diarrhea, nausea, vomiting, and hypokalemia [3]. Less common but serious side effects can also occur [3]. Patients should discuss potential risks and benefits with their healthcare provider [3].
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Sources:
[1] https://www.ema.europa.eu/en/medicines/human/EPAR/defitelio
[2] https://www.jazzpharma.com/pipeline/pipeline-overview/defitelio/
[3] https://www.ema.europa.eu/en/documents/overview-scientific-advice/oral-explanation-defitelio-scientific-advice-pediatric-investigation-plan-protocol-amendment-3-day-1-2215378_en.pdf
[4] https://www.jazzpharma.com/our-products/defitelio/