Application Detail

Description of Medical Service

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.

Description of Medical Condition

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.

Reason for Application

Referral from the National Blood Authority

Medical Service Type

Therapeutic technology

Previous Application Number/s

Not Applicable

Associated Documentation

Application Summary and PICO Set

Application Summary (PDF 405 KB)
Application Summary (Word 28 KB)

PICO Set 1 (PDF 1446 KB)
PICO Set 1 (Word 232 KB)

PICO Set 2 (PDF 1445 KB)
PICO Set 2 (Word 417 KB)

Consultation Survey

Consultation Survey (PDF 651 KB)
Consultation Survey (Word 32 KB)

PASC Consultation
PASC consultation closes 15 March 2024

MSAC Consultation
MSAC consultation TBA – please subscribe to the MSAC Email Bulletin to be notified when consultation opens for this application

For further information please refer to PASC, ESC, MSAC Key Dates
For further information on the consultation process please refer to MSAC Consultation Process

PICO Confirmation

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Assessment Report

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Public Summary Document

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Meetings for this Application

PASC

18–19 April 2024

ESC

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MSAC

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