The European Haemophilia Consortium (EHC) has published the second issue (2019 Issue 1) of its ‘Novel treatments in haemophilia and other bleeding disorders: A periodic EHC review.

In this issue we review any developments in novel technologies in haemophilia and other rare bleeding disorders that occurred since May 2018, when the first issue was published.

Like the first issue, the information provided in this newsletter was compiled from multiple sources such as presentations and abstracts from recent scientific meetings, websites (for e.g. clinicaltrial.gov) and by writing directly to pharmaceutical companies.

The content was then redrafted in an easy-to-understand language by the EHC New Products Committee.

Here below you will find a preview of the content in this issue:

Extended half-life

  • Jivi granted marketing authorisation
  • An update on clinical studies and real-world data on switching patients from SHL FVIII to EHL FVIII
  • New EHL FVIII announced at ASH

Non-replacement therapies

  • Hemlibra® (emicizumab)
    • An update on NIS and HAVEN 3 & 4 studies
  • Fitusiran
    • An update on fitusiran
    • An update on anti-TFPI BAY1093884

Gene therapy

  • An update on FVIII gene therapy clinical trials with valoctocogene roxaparvovec (BMN270)
  • An update on FVIII gene therapy clinical trial with SPK-8011
  • An update on GO-8 FVIII gene therapy clinical trial
  • An update on the ALTA studies
  • Get8 trial is underway
  • Pre-clinical developments of AMT-180 for gene therapy in haemophilia A with and without inhibitors

Extended half-life

  • An update on clinical studies and real world data on switching patients from SHL FIX to EHL FIX
  • An update on SQ FIX

Non-replacement therapies

  • Fitusiran

Gene therapy

  • An update on HOPE B FIX gene therapy studies
  • An update on fidanacogene elaparvovec (SPK-9001) FIX gene therapy trial
  • An update on FIX gene therapy trial with scAAV2/8-LP1-hFIXco
  • An update on FLT180a FIX gene therapy trial
  • An update on SB FIX genome editing trial

Extended half-life

  • An update on MarzAA
  • Clinical trial for rFVIIa-FP is terminated

Non-replacement therapies

  • Fitusiran
  • Hemlibra® (emicizumab)
    • An update on HAVEN 2 study

Gene therapy

  • Clinical trial to start for gene therapy study in people with haemophilia A and inhibitors
  • Pre-clinical development of AMT-180 for gene therapy in haemophilia A with and without inhibitors
  • Novo Seven® receives license expansion for Glanzmann’s thrombasthenia
  • Post-marketing studies for Obizur®
  • Development of APC inhibitor
  • IDO 8 granted orphan drug designation in the EU and US
  • Development in cell therapy for haemophilia