The Supply of a licensed factor IX gene therapy
Buyer: St James's Hospital
- Published
- 1 April 2025
- Procedure
- Negotiated procedure
- Lots
- 1
- Notice number
- 00209093-2025
- Reference
- e15b99ea-2c91-43b2-aa03-f6b6c4317bcf
- Official source
- Official source
CPV codes
- 33621000Medicamentos para la sangre y para los órganos hematopoyéticos
Description
, people with severe or moderately-severe factor IX deficiency are at risk of severe, life threatening bleeding unless factor IX is replaced. The current method of treatment is to replace factor IX using factor IX clotting factor concentrate which is given intravenously on a regular weekly schedule (prophylaxis). The current standard of care for people with severe or moderately-severe factor IX deficiency in Ireland is that all patients are treated with factor IX prophylaxis. Despite regular prophylaxis, certain patients do not achieve adequate bleed control and require increased amounts of FIX replacement leading to higher-than-average FIX consumption. In addition, some patients have difficulty with venous access meaning that an intravenous treatment is not suitable. Factor IX gene therapy has been developed and licensed to address these needs by providing a continuous source of factor IX through transfection of the patient’s own liver cells, generally with much higher trough factor IX levels than would be achieved with prophylaxis. There is a clinical need for certain patients to have access to factor IX gene therapy to provide better bleed protection and reduce factor IX concen…
Official publications
- TED · 00209093-2025 · 1 April 2025
- OJS · 64/2025 · 1 April 2025
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