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defibrotide

Defitelio · ATC code B01AX01

Also known as: Defibrotid

Key facts
Active substance
defibrotide
Drug class
Antithrombotic
ATC code
B01AX01
Authorisation
centrally authorised across the EU (EMA)
Dosage forms
Defitelio 80 mg/mL concentrate for solution for infusion
Marketing authorisation holder
Gentium S.r.l.
Information as of
2026-10-29

Key facts

Defibrotide (Defitelio) is an antithrombotic and profibrinolytic agent used to treat severe hepatic veno-occlusive disease after haematopoietic stem-cell transplantation.

What it is used for

  • Treatment of severe hepatic veno-occlusive disease (VOD), also known as sinusoidal obstruction syndrome (SOS), in haematopoietic stem-cell transplantation therapy, in adults and in adolescents, children and infants over 1 month of age.

Dosing

  • Adults and children aged 1 month to 18 years: 6.25 mg/kg body weight every 6 hours (25 mg/kg/day) as a 2-hour intravenous infusion; doses above 25 mg/kg/day are not recommended.
  • Treatment lasts at least 21 days and continues until signs and symptoms of severe VOD resolve.
  • Always diluted before use (5% glucose or sodium chloride solution) to a final concentration of 4 mg/mL to 20 mg/mL.
  • No dose adjustment in renal impairment or intermittent haemodialysis; no adjustment but careful monitoring in hepatic impairment.
  • Not recommended in children under one month.

Contraindications

  • Hypersensitivity to the active substance or to any of the excipients.
  • Concomitant thrombolytic therapy (e.g. t-PA).

Important warnings

  • Medicines increasing bleeding risk are not recommended within 24 hours of administration (within 12 hours for unfractionated heparin).
  • Concomitant systemic anticoagulants require careful monitoring; platelet aggregation inhibitors such as NSAIDs should be given with care.
  • Not recommended or to be stopped in clinically significant acute bleeding requiring transfusion; temporary discontinuation before surgery or invasive procedures with major bleeding risk.
  • Not recommended in haemodynamic instability (inability to maintain mean arterial pressure with single pressor support).
  • Bolus administration may cause flushing or a sensation of generalised heat.

Side effects

  • Very common: hypotension.
  • Common: haemorrhage (including gastrointestinal, pulmonary and cerebral haemorrhage, epistaxis, haematuria, catheter site haemorrhage), coagulopathy, vomiting, diarrhoea, nausea, rash, pruritus, petechiae, pyrexia.
  • Uncommon: hypersensitivity, anaphylactic reaction, cerebral haematoma, haemothorax, melaena.

Interactions

  • Recombinant t-PA may increase the risk of haemorrhage; co-administration is contraindicated.
  • Use with antithrombotic/fibrinolytic medicines (e.g. low molecular weight heparins, warfarin, dabigatran, rivaroxaban, apixaban) is not recommended.

Pregnancy and breastfeeding

  • Should not be used during pregnancy unless the woman's condition requires it; effective contraception for patients and partners during treatment and for one week after.
  • May be used during breast-feeding.

Summary of the official product information, written with AI on 2026-10-29. Every number was checked automatically against the original text. Not reviewed by a physician – the full official text is authoritative.

Full official text →

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For patients

Package leaflet, verbatim

This product has no separate US patient information.

Authorisation and brand names by country

Medicines with exactly this active-substance composition, as listed in the national registers – alphabetical and without ranking. Whether a product is currently available is not shown here.

EU – centrally authorised (all member states)

Source: European Medicines Agency (EMA), product information

Brand names in 3 more European countries
Iceland
Defitelio
Liechtenstein
Defitelio
Norway
Defitelio

EMA – Article 57 product data (EU/EEA), 2026-10-29

Medelix receives no money from pharmaceutical companies. Brand names are listed alphabetically and without ranking.

Other substances in the same group

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Sources

  1. European Medicines Agency. Defitelio – EPAR, Product Information. 2026-10-29. https://www.ema.europa.eu/en/documents/product-information/defitelio-epar-product-information_en.pdf
  2. Wikidata (CC0): ATC B01AX.

Editorial: Medelix editorial team · last updated 2026-10-29 · How these pages are made · Report an error

Source: European Medicines Agency (EMA), product information. Not advertising, not a treatment recommendation. For questions about your own treatment, ask your doctor or pharmacist.