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.
Treatment by Cohort
Download entire data set here
Products
1. Veyvondi®/Vonvendi (vonicog alfa), recombinant
2. Wilfactin (VWF), plasma-derived
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
Products
1. Octostim/Emosint/ Minirin/Stimate
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-redived
15. Aafact®, plasma-redived
16. Amofil, plasma-redived
17. Emoclot/Klott/Emowil, plasma-redived
18. Beriate, plasma-redived
19. Immunate/Immunate SD, plasma-redived
20. Alphanate®/Fanhdi, plasma-redived
21. Talate, plasma-redived
22. 8Y, plasma-redived
23. Factane, plasma-redived
24. Haemoctin/Haemoctin Sdh, plasma-redived
25. Humaclot/Humafactor-8, plasma-redived
26. Koate DVI, plasma-redived
27. Voncento®/Haemate-P/Humate-P/Haemate, plasma-redived
28. Wilate/Wilnativ, plasma-redived
29. Optivate/Optiwate, plasma-redived
30. Octafil/Octafil LV, plasma-redived
*Licensed in all EU countries
Products
1. Hemlibra
*Licensed in all EU countries
Products
1. RoctavianTM (valoctogene roxaparvovec)
Products
1. Octostim / Emosint / Minirin
Products
1. Cevenfacta® (eptacog beta), recombinant
2. NovoSeven®/NovoSeven® RT (eptacog alfa), recombinant
3. FEIBA (activated prothrombin complex concentrate), plasma-derived
Products
1. Hemlibra
*Licenced in all EU countries
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
Products
1. Alhemo (concizumab)
Products
1. Hemgenix® (etranacogene dezaparvovec)
Products
1. Cevenfacta® (eptacog beta), recombinant
2. NovoSeven®/NovoSeven® RT (eptacog alfa), recombinant
3. FEIBA (activated prothrombin complex concentrate), plasma-derived
Products
1. CLOTTAFACT®/Fibriclotte, plasma-derived
2. Riastap/Haemocomplettan, plasma-derived
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
Products
1. Octaplas (virus-inactivated fresh frozen plasma)
Products
1. NovoSeven/NovoSeven RT (eptacog alfa), recombinant
2. Provertin-Um TIM3, plasma-derived
Products
1. Coagadex, plasma-derived
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
Products
1. Hemoleven, complasma-derived
Products
1. NovoThirteen®/ Tretten, recombinant*
2. Cluviat/Fibrogammin, complasma-derived
*Licenced in all EU countries
Products
1. NovoSeven/NovoSeven RT (eptacog alfa), recombinant
Products
1. Tranex/Ugurol
Products
1. Cyklokapron/Caprolisin
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.
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
The EHC herewith communicates the following:
Full information can be found here https://www.ehc.eu/baxject-ii-baxject-ii-hi-flow-reconstitution-devices/
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
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