The Supply of a licensed factor IX gene therapy

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 …

CPV: 33620000 Medicamentos para la sangre, para los órganos hematopoyéticos y para el aparato cardiovascular, 33621000 Medicamentos para la sangre y para los órganos hematopoyéticos
Lugar de ejecución:
The Supply of a licensed factor IX gene therapy
Organismo adjudicador:
St James's Hospital
Número de premio:
1

1. Buyer

1.1 Buyer

Official name : St James's Hospital
Legal type of the buyer : Body governed by public law
Activity of the contracting authority : Health

2. Procedure

2.1 Procedure

Title : The Supply of a licensed factor IX gene therapy
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 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. The contracting authority intends to award a control to CSL Behring GmbH for 3 years for product for its licensed Factor IX Gene Therapy Hemgenix (etranacogene dezaparovec-drib)
Procedure identifier : fd4b3d0a-f13e-4968-9337-abf465616e2d
Type of procedure : Negotiated without prior call for competition

2.1.1 Purpose

Main nature of the contract : Supplies
Main classification ( cpv ): 33620000 Medicinal products for the blood, blood-forming organs and the cardiovascular system

2.1.4 General information

Legal basis :
Directive 2014/24/EU

5. Lot

5.1 Lot technical ID : LOT-0001

Title : The Supply of a Factor IX Gene Therapy Product
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 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. The contracting authority intends to award a control to CSL Behring GmbH for 3 years for product for its licensed Factor IX Gene Therapy Hemgenix (etranacogene dezaparovec-drib)
Internal identifier : 1

5.1.1 Purpose

Main nature of the contract : Supplies
Main classification ( cpv ): 33621000 Medicinal products for the blood and blood-forming organs

5.1.6 General information

Procurement Project not financed with EU Funds.
The procurement is covered by the Government Procurement Agreement (GPA) : no

5.1.7 Strategic procurement

Aim of strategic procurement : No strategic procurement

5.1.16 Further information, mediation and review

Review organisation : The High Court of Ireland -

6. Results

Direct award :
Justification for direct award : The contract can be provided only by a particular economic operator because of exclusive rights, including intellectual property rights
Other justification : 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. The contracting authority intends to award a control to CSL Behring GmbH for 3 years for product for its licensed Factor IX Gene Therapy Hemgenix (etranacogene dezaparovec-drib)

6.1 Result lot ldentifier : LOT-0001

6.1.2 Information about winners

8. Organisations

8.1 ORG-0001

Official name : St James's Hospital
Registration number : St James Hospital
Postal address : James Street
Town : Dublin
Postcode : D08 NHY1
Country subdivision (NUTS) : Dublin ( IE061 )
Country : Ireland
Telephone : 014284707
Internet address : https://www.stjames.ie/
Roles of this organisation :
Buyer

8.1 ORG-0002

Official name : The High Court of Ireland
Registration number : The High Court of Ireland
Department : The High Court of Ireland
Postal address : Four Courts, Inns Quay, Dublin 7
Town : Dublin
Postcode : D07 WDX8
Country subdivision (NUTS) : Dublin ( IE061 )
Country : Ireland
Telephone : +353 1 8886000
Roles of this organisation :
Review organisation

8.1 ORG-0003

Official name : European Dynamics S.A.
Registration number : 002024901000
Department : European Dynamics S.A.
Town : Athens
Postcode : 15125
Country subdivision (NUTS) : Βόρειος Τομέας Αθηνών ( EL301 )
Country : Greece
Telephone : +30 2108094500
Roles of this organisation :
TED eSender
Notice information
Notice identifier/version : 6caef4d0-ad06-47de-94ec-0955c37b0a30 - 01
Form type : Direct award preannouncement
Notice type : Voluntary ex-ante transparency notice
Notice dispatch date : 28/03/2025 17:42 +00:00
Languages in which this notice is officially available : English
Notice publication number : 00208269-2025
OJ S issue number : 64/2025
Publication date : 01/04/2025