Orphan designation: Adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a synthetic hepatic combinatorial bundle promoter Treatment of haemophilia A,…
This medicine, also known as ASC618, is made of a virus that has been modified to contain a gene that encodes for factor VIII, the protein that is lacking in patients with haemophilia A. This protein is necessary for the formation of blood clots. When ASC618 is given to the patient, the modified virus is expected to carry the gene into the liver cells, enabling them to produce factor VIII. It is expected that a single dose of the medicine will elevate levels of factor VIII for a long period, thereby helping the blood to clot and controlling bleeding. The type of virus used in this medicine ('adeno-associated virus') does not cause disease in humans.
Based on description provided by sponsor
Stage of development at time of orphan designation
At the time of submission of the application for orphan designation:
- The effects of the medicine had been evaluated in experimental models.
- No clinical trials with the medicine in patients with haemophilia A had been started.
More information on how potential new medicines are tested during their development is available on Authorisation of medicines.
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