Airija – Kraują ir kraujodarą veikiantys vaistai – The Supply of a licensed factor IX gene therapy

Airija – Kraują ir kraujodarą veikiantys vaistai – The Supply of a licensed factor IX gene therapy

I dalis: Perkančioji organizacija

I.1) Pavadinimas ir adresai:

Oficialus pavadinimas: St James's Hospital
Adresas: James Street
Miestas: Dublin
Pašto kodas: D08 NHY1
Šalis: Airija
Asmuo ryšiams:
El-paštas: dgunning@stjames.ie
Interneto adresas (-ai):
Pagrindinis adresas: https://www.stjames.ie/

II dalis: Objektas

II.1.1) Pavadinimas:

The Supply of a licensed factor IX gene therapy

II.1.2) Pagrindinis BVPŽ kodas:

33621000 Kraują ir kraujodarą veikiantys vaistai

II.1.3) Sutarties tipas:

Kita

II.1.4) Trumpas aprašymas:

, 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 concentrate consumption. The factor IX gene therapy is an advanced therapeutic medicinal product which would replace the need for factor IX clotting factor concentrate for prophylaxis in treated patients.

II.2) Aprašymas:

II.2.1) Kitas (-i) šio pirkimo BVPŽ kodas (-ai):

33621000 Kraują ir kraujodarą veikiantys vaistai