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Defitelio

31 July 2020

The Australian Prescription Medicine Decision Summary provides a short overview of the TGA's evaluation process leading to the registration of a new prescription medicine on the Australian Register of Therapeutic Goods (ARTG).

More in-depth information about the evaluation will be available in the Australian Public Assessment Report (AusPAR) for a particular prescription medicine, which can be found on the AusPAR search page once published.

Australian prescription medicine decision summary

Summary of submission

Submission type
New biological entity
Product name
Defitelio
Active ingredients
Defibrotide
ATC codes
B01AX01
Decision
Approved
Date of decision
15 July 2020
Date of entry onto ARTG
23 July 2020
ARTG numbers
319221
Black Triangle Scheme
Yes. This product will remain in the scheme for 5 years, starting on the date the product is first supplied in Australia
Sponsor
Link Medical Products Pty Ltd
Sponsor address
5 Apollo St, Warriewood, NSW 2102
Dose forms
Concentrated solution for infusion
Strength
200 mg/2.5 mL
Other ingredients
Sodium citrate dihydrate, hydrochloric acid (for pH adjustment), sodium hydroxide (for pH adjustment), water for injections
Containers
Vial
Pack sizes
10
Routes of administration
Intravenous infusion
Dosage

Defitelio must be prescribed and administered to patients by specialised physicians experienced in the diagnosis and treatment of complications of haematopoietic stem-cell transplantation (HSCT).

The recommended dose is 6.25 mg/kg body weight every 6 hours (25 mg/kg/day).

There is limited efficacy and safety data on doses above this level and consequently it is not recommended to increase the dose above 25 mg/kg/day.

The treatment should be administered for a minimum of 21 days and continued until the symptoms and signs of severe veno‑occlusive disease (VOD) resolve.

For further information refer to the Product Information.

Pregnancy category

D

Drugs which have caused, are suspected to have caused or may be expected to cause, an increased incidence of human fetal malformations or irreversible damage. These drugs may also have adverse pharmacological effects. Accompanying texts should be consulted for further details.

The use of any medicine during pregnancy requires careful consideration of both risks and benefits by the treating health professional. This must not be used as the sole basis of decision making in the use of medicines during pregnancy. The TGA does not provide advice on the use of medicines in pregnancy for specific cases. More information is available from obstetric drug information services in your State or Territory.

What was approved?

Defitelio (defibrotide) was approved for the following therapeutic use:

Defitelio is indicated for the treatment of severe hepatic veno-occlusive disease (VOD) also known as sinusoidal obstruction syndrome (SOS) in haematopoietic stem-cell transplantation (HSCT) therapy.

It is indicated in adults and in adolescents, children and infants of 1 month of age and above.

What is this medicine and how does it work?

What was the decision based on?

What steps were involved in the decision process?

What post-market commitments will the sponsor undertake?

  • Defitelio (defibrotide) is to be included in the Black Triangle Scheme. The Product Information (PI) (including the PI as package insert) and the Consumer Medicines Information (CMI) for Defitelio must include the black triangle symbol and mandatory accompanying text for five years, which starts from the date that the sponsor notifies the TGA of supply of the product.
  • The Defitelio European Union-Risk Management Plan (EU-RMP; version 7.0, dated 19 December 2019, data lock point 18 October 2019), with Australian specific Annex (version 1.0, dated June 2020), included with submission PM-2019-01763-1-3, and any subsequent revisions, as agreed with the TGA will be implemented in Australia.

    An obligatory component of risk management plans is routine pharmacovigilance. Routine pharmacovigilance includes the submission of periodic safety update reports (PSURs).

    Reports are to be provided in line with the current published list of European Union reference dates and frequency of submission of PSURs until the period covered by such reports is not less than three years from the date of the approval letter.

  • All batches of Defitelio (defibrotide) imported into Australia must comply with the product details and specifications approved during evaluation and detailed in the Certified Product Details (CPD).
  • Up to five (5) initial batches of Defitelio (defibrotide) imported into Australia is not released for sale until samples and/or the manufacturer's release data have been assessed and endorsed for release by the TGA Laboratories Branch. Outcomes of laboratory testing are published biannually in the TGA Database of Laboratory Testing Results.

    The sponsor should be prepared to provide product samples, reference materials and documentary evidence as defined by the TGA Laboratories branch. The sponsor must contact Biochemistry.Testing@health.gov.au for specific material requirements related to the batch release testing/assessment of the product. More information is available at Testing of biological medicines

    This batch release condition will be reviewed and may be modified on the basis of actual batch quality and consistency. This condition remains in place until the sponsor is notified in writing of any variation.

  • Certified Product Details

    The CPD, as described in Guidance 7: Certified Product Details of the Australian Regulatory Guidelines for Prescription Medicines (ARGPM), in PDF format, for the products should be provided upon registration of these therapeutic goods. In addition, an updated CPD should be provided when changes to finished product specifications and test methods are approved in a Category 3 application or notified through a self-assessable change.

    CPDs should be emailed to Biochemistry.Testing@health.gov.au as a single PDF document.

  • For all injectable products the Product Information must be included with the product as a package insert.

Further information

The latest Product Information (PI) and Consumer Medicines Information (CMI) can be found at: ARTG search.

Australian Public Assessment Reports (AusPARs) can be found at: AusPAR search.

The latest news and updates regarding therapeutic goods regulation can be found at: TGA news room.