Treatment

From the detailed information about how blood clots and how each treatment option works in simple words and different multimedia formats in EHCucate to a cohort-based approach towards licenced treatment and scientific updates on the novel treatments in development in the Novel Treatment Review created by the EHC for its community.

Treatment options

There are many treatment options either already marketed and available for bleeding disorders or at different stages of the drug development process. The EHC Academy builds upon the latest scientific discoveries and approved approaches in the scientific community.    

Learn about all treatment options in our EHCucate project and create your learning path through interaction with different multimedia formats!

Access a cohort-based approach to the licenced-in-the-EU treatment options, or get to know the latest updates in the novel treatments, including the drugs in development, in the periodic Novel Treatment Review.  

The EHC greatly welcomes all treatment developments that may benefit patients in the future. The EHC takes no position on any product type or class reported in this newsletter.

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Treatment by Cohort
Download entire data set here

Von Willebrand Disease (VWD)
VWF concentrates

Products

1. Veyvondi®/Vonvendi (vonicog alfa), recombinant
2. Wilfactin (VWF), plasma-derived

Von Willebrand Disease (VWD)
VWF/FVIII concentrates

Products

1. Voncento®/Haemate-P/Humate-P (VWF/FVIII), plasma-derived *
2. Fanhdi/Alphanate (VWF/FVIII), plasma-derived
3. Wilate, (VWF/FVIII), plasma-derived
4. Immunate/Immunate Stim Plus, (VWF/FVIII), plasma-derived

*Licensed in all EU countries

Von Willebrand Disease (VWD)
Desmopressin

Products

1. Octostim/Emosint/ Minirin/Stimate

Haemophilia A without Inhibitors
FVIII concentrates

Products

1. Advate® (octocog alfa), recombinant
2. Adynovi®/Adynovate®(rurioctocog alfa pegol), recombinant
3. Afstyla® (lonoctocog alfa), recombinant*
4. Elocta®/Eloctate® (efmoroctocog alfa), recombinant
5. Esperoct® (turoctocog alfa pegol), recombinant
6. Jivi® (damoctocog alfa pegol), recombinant*
7. Kogenate® FS (octocog alfa), recombinant
8. Kogenate Bayer/Helixate NexGen/Iblias (octocog alfa), recombinant
9. Kovaltry® (octocog alfa), recombinant*
10. Novoeight® (turoctocog alfa), recombinant*
11. Nuwiq®/Vihuma(simoctocog alfa), recombinant
12. Refacto AF® (moroctocog alfa), recombinant
13. Recombinate (octocog alfa), recombinant
14. Octanate/Octanate kons/Octanate LV, plasma-derived
15. Aafact®, plasma-derived
16. Amofil, plasma-derived
17. Emoclot/Klott/Emowil, plasma-rerived
18. Beriate, plasma-derived
19. Immunate/Immunate SD, plasma-derived
20. Alphanate®/Fanhdi, plasma-derived
21. Talate, plasma-derived
22. 8Y, plasma-derived
23. Factane, plasma-derived
24. Haemoctin/Haemoctin Sdh, plasma-derived
25. Humaclot/Humafactor-8, plasma-derived
26. Koate DVI, plasma-derived
27. Voncento®/Haemate-P/Humate-P/Haemate, plasma-derived
28. Wilate/Wilnativ, plasma-derived
29. Optivate/Optiwate, plasma-derived
30. Octafil/Octafil LV, plasma-derived

*Licensed in all EU countries

Haemophilia A without Inhibitors
FVIII mimetic/ Bispecific antibody

Products

1. Hemlibra

*Licensed in all EU countries

Haemophilia A without Inhibitors
Gene Therapy

Products

1. RoctavianTM (valoctogene roxaparvovec)

Haemophilia A without Inhibitors
Desmopressin

Products

1. Octostim / Emosint / Minirin

Haemophilia A with Inhibitors
Bypassing agent

Products

1. Cevenfacta® (eptacog beta), recombinant
2. NovoSeven®/NovoSeven® RT (eptacog alfa), recombinant
3. FEIBA (activated prothrombin complex concentrate), plasma-derived

Haemophilia A with Inhibitors
FVIII mimetic/ Bispecific antibody

Products

1. Hemlibra

*Licenced in all EU countries

Haemophilia B
FIX concentrates

Products

1. Alprolix® (eftrenonacog alfa), recombinant
2. BeneFIX® (nonacog alfa), recombinant
3. Idelvion® (albutrepenonacog alfa), recombinant*
4. Refixia®/Rebinyn® (nonacog beta pegol), recombinant
5. Rixubis® (nonacog gamma), recombinant
6. Immunine/Immunine Stim Plus, plasma-derived
7. Octanine, plasma-derived
8. BETAFACT®, plasma-derived
9. AimaFIX®/IXED, plasma-derived
10. Alphanine, plasma-derived

*Licenced in all EU countries

Haemophilia B
anti-TFPI antibodies

Products

1. Alhemo (concizumab)

Haemophilia B
Gene Therapy

Products

1. Hemgenix® (etranacogene dezaparvovec)

Haemophilia B with Inhibitors
Bypassing agent

Products

1. Cevenfacta® (eptacog beta), recombinant
2. NovoSeven®/NovoSeven® RT (eptacog alfa), recombinant
3. FEIBA (activated prothrombin complex concentrate), plasma-derived

Fibrinogen disorders
Fibrinogen concentrates

Products

1. CLOTTAFACT®/Fibriclotte, plasma-derived
2. Riastap/Haemocomplettan, plasma-derived

Factor II (Prothrombin) Deficiency
Prothrombin complex concentrate

Products

1. Uman Complex D.I./Kedcom, plasma-derived
2. Prothromplex TIM 3/Proplex, plasma-derived
3. Confidex, plasma-derived
4. Beriplex, plasma-derived
5. Octaplex, plasma-derived

Factor V Deficiency
No factor concentrate available

Products

1. Octaplas (virus-inactivated fresh frozen plasma)

Factor VII Deficiency
FVII concentrate

Products

1. NovoSeven/NovoSeven RT (eptacog alfa), recombinant
2. Provertin-Um TIM3, plasma-derived

Factor X Deficiency
FX concentrate

Products

1. Coagadex, plasma-derived

Factor X Deficiency
Prothrombin complex concentrate

Products

1. Uman Complex D.I./Ked, complasma-derived
2. Prothromplex TIM 3/Proplex, complasma-derived
3. Confidex, complasma-derived
4. Beriplex, complasma-derived
5. Octaplex, complasma-derived

Factor XI Deficiency
FXI concentrate

Products

1. Hemoleven, complasma-derived

Factor XIII Deficiency
FXIII concentrate

Products

1. NovoThirteen®/ Tretten, recombinant*
2. Cluviat/Fibrogammin, complasma-derived

*Licenced in all EU countries

Glanzmann’s Thromboasthenia
Activated FVII concentrate

Products

1. NovoSeven/NovoSeven RT (eptacog alfa), recombinant

Antifibrinolytic Agents
Tranexamic Acid

Products

1. Tranex/Ugurol
2. Cyklokapron

Antifibrinolytic Agents
Aminocaproic Acid

Products

1. Caprolisin

Antihemorrhagic Agents
Sulfonic Acid

Products

1. Etamsylate

EHCucate

Education on all treatment options tailored to you!

EHCucate is an educational project on therapies of the EHC. It was launched as an app in October 2021 and is now available on both iOS and Android platforms.

The bleeding disorders community has diverse learning needs. And EHCucate addresses these needs. It is designed to allow learners to choose the content they interact with and how the interaction happens, reassured that the source information comes from is impartial, trustworthy, and reliable.

Start Learning

Download the app on your mobile phone to get accurate, unbiased, and up-to-date information about therapies for inherited bleeding disorders and share it with your community.

Everything you need to know about rare bleeding disorders in your pocket, in any format and always up to date. Only trustworthy, unbiased, and verified information.

31 May 2024
EMA recommends granting a conditional marketing authorisation for Durveqtix (fidanacogene elaparvovec)

EMA’s human medicines committee (CHMP) recommended eight medicines for approval at its May 2024 meeting.

A recommendation for a conditional marketing authorisation was made for Durveqtix (fidanacogene elaparvovec), a new gene therapy treatment for haemophilia B, a rare inherited bleeding disorder. This medicine was supported through EMA’s Priority Medicines (PRIME) scheme, which provides early and enhanced scientific and regulatory support for promising medicines with a potential to address unmet medical needs. See more details in the news announcement in the grid below.

More information here

26 April 2024
EMA recommends granting a marketing authorisation for Altuvoct* (efanesoctocog alfa)

EMA’s human medicines committee (CHMP) recommended eight medicines for approval at its April 2024 meeting.

The CHMP recommended granting a marketing authorisation for Altuvoct* (efanesoctocog alfa), for the treatment and prophylaxis of bleeding in patients with haemophilia A, a rare inherited bleeding disorder caused by lack of factor VIII.

More information here

August 2023
Baxject II/Baxject II Hi-Flow Reconstruction Devices – Informational Communication and EHC Statement

The EHC herewith communicates the following:

  • There has been a small number of complaints for the BAXJECT II and BAXJECT II Hi-Flow reconstitution devices that concern the presence of particulate matter before administration.
  • This issue is not related with any medicine produced by Takeda (RECOMBINATE, RIXUBIS, ADVATE, ADYNOVATE and FEIBA) or with the diluent used for the reconstitution of these products, but with the device (BAXJECT II) that is used to mix the medicine and diluent, before administration.
  • There hasn’t been any presence of particles in the medicine/diluent reported.
  • These devices are co-packaged with the medicine and the diluent.
  • No adverse events linked to the particulate matter have been reported.
  • Takeda has decided, as a precautionary measure, to voluntarily replace the devices manufactured between October 2021 and January 2022.

Full information can be found here https://www.ehc.eu/baxject-ii-baxject-ii-hi-flow-reconstitution-devices/

February 2023
Hemgenix receives a conditional marketing authorisation valid throughout the EU

The first gene therapy for haemophilia B has been given conditional marketing authorisation in Europe by the European Commission on February 20, 2023. 

Hemgenix is a medicine used to treat adults with severe and moderately severe haemophilia B, an inherited bleeding disorder caused by the lack of factor IX (a protein needed to produce blood clots to stop bleeding). It is used in adults who have not developed inhibitors (proteins made by the body’s natural defenses) against factor IX.

Haemophilia B is rare, and Hemgenix was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 21 March 2018. Further information on the orphan designation can be found here: EU/3/18/1999 

Hemgenix contains the active substance etranacogene dezaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

Read more on the EMA website:  https://www.ema.europa.eu/en/medicines/human/EPAR/hemgenix#:~:text=information%20on%20Hemgenix-,Overview,blood%20clots%20to%20stop%20bleeding

August 2022
Roctavian received a conditional marketing authorisation valid throughout the EU

Roctavian is a medicine for treating severe haemophilia A, an inherited bleeding disorder caused by the lack of a clotting protein known as factor VIII. It is used in adults who do not have inhibitors (antibodies) against factor VIII and who have no antibodies against adeno-associated virus serotype 5 (AAV5). Roctavian contains the active substance valoctocogene roxaparvovec and is a type of advanced therapy medicine called a ‘gene therapy product’. This is a type of medicine that works by delivering genes into the body.

 

Read more on the EMA website: 

https://www.ema.europa.eu/en/documents/overview/roctavian-epar-medicine-overview_en.pdf

Treatment Updates

In this section periodic updates will be provided to inform the bleeding disorders community about product updates, related safety issues, licensing updates, etc.

Novel Treatment Product Reviews

2024

Issue One

The EHC encourages its NMOs to use the information contained in this review at a national level between their patients, health professionals and other relevant stakeholders but takes no responsibility for any changes.

2022

Issue Two

We primarily cover news from the 2022 Congress of the World Federation of Hemophilia (WFH), held in May 2022, and the 2022 Congress of the International Society for Thrombosis and Haemostasis (ISTH), held in July 2022

2022

Issue One

We primarily cover news from the 2022 virtual American Society of Hematology (ASH), held in December 2021, and the 2022 virtual Conference of the European Association for Haemophilia and Allied Disorders (EAHAD), held in February 2022