Applications

1751 – Valoctocogene roxaparvovec gene therapy for congenital haemophilia A

Find out about the service or technology in this application and the medical condition it addresses. You can also view the application documents, the deadlines for providing consultation input and the outcome of the application when the MSAC process is complete.

Application details

Reason for application

Referral from the National Blood Authority

Service or technology in this application

Valoctocogene roxaparvovec is a gene therapy that uses an adeno-associated virus type 5 (AAV5), to deliver a healthy copy of the F8 gene. Following infusion with valoctocogene roxaparvovec into haemophilia A patients, the recombinant F8 gene is delivered to cell nuclei of several tissues. As the new F8 gene is under the control of a liver-specific promoter, only liver cells are able to activate transcription to make FVIII protein which is then secreted into the bloodstream. As a gene therapy, valoctocogene roxaparvovec is provided as a single, one-time intravenous infusion.

Type: Therapeutic technology

Medical condition this application addresses

Haemophilia A is genetic condition where a variation in the FVIII (F8) gene results in a deficiency of the coagulation factor VIII (FVIII). Affected individuals have an increased tendency to bleed, with severity of bleeding directly correlated to the level of clotting factor. In the most severe cases, bleeding episodes most commonly occur internally into the joints or muscles leading to painful, progressive and irreversible joint damage, however some bleeds can be life threatening, such as intracranial or gastrointestinal bleeds. Bleeding may occur spontaneously without trauma or injury.

Application documents

Application summary

PICO set 1

PICO set 2

Consultation survey

PICO confirmation

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Consultation survey and deadlines

  • PASC consultation: Closed
  • MSAC consultation: To be confirmed – please subscribe to the MSAC email bulletin to be notified when this application is scheduled for MSAC consideration. The bulletin will also advise the closing date for consultation input.

Find out more about the consultation process.

Meetings to consider this application

  • PASC meeting: 18–19 April 2024
  • ESC meeting: To be confirmed
  • MSAC meeting: To be confirmed