Haemophilia

In the In Conversation With series, a part of the free DDW podcast, DDW speaks with members of the drug discovery industry about their work and how it helps turn science into business.    In this episode, Megan Thomas is in conversation with Dr Jude Samulski, Co-founder and Chief Scientific Officer at AskBio. Dr. Samulski’s graduate work demonstrated the […]

In a draft guidance consultation, the UK National Institute for Health and Care Excellence (NICE) has decided not to recommend the use of Hemgenix (etranacogene dezaparvovec) on the NHS for treating haemophilia B (congenital factor IX deficiency). Although the gene therapy was granted a marketing authorisation by the UK Medicines and Healthcare products Regulatory Agency […]

The US Food and Drug Administration (FDA) has approved the first gene therapy for adults with severe haemophilia A. BioMarin’s Roctavian (valoctocogene roxaparvovec-rvox) has been approved for the treatment of adults with severe haemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected […]

CSL’s Hemgenix (etranacogene dezaparvovec) has been authorised as the first gene therapy for adults with severe and moderately severe haemophilia B (congenital Factor IX (FIX) deficiency) in Great Britain. It has been granted a marketing authorisation for patients without a history of FIX inhibitors by the UK Medicines and Healthcare products Regulatory Agency (MHRA). The […]

The European Commission has granted conditional marketing authorisation for CSL’s Hemgenix (etranacogene dezaparvovec), the first one-time gene therapy for severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adults without a history of Factor IX inhibitors.   In an ongoing clinical trial, the drug reduced the rate of annual bleeds with a single infusion […]

An investigational one-time gene therapy for the treatment of adults with severe haemophilia A has been proven to be effective for more than three years from treatment.  BioMarin Pharmaceutical revealed the positive results from its ongoing global Phase III GENEr8-1 study of Roctavian (valoctocogene roxaparvovec).   This is the largest and longest global Phase III study […]

The first gene therapy for haemophilia B could soon be available to patients in Europe following a positive opinion from the Committee for Medicinal Products for Human Use (CHMP).  The CHMP has recommended conditional marketing authorisation of CSL’s etranacogene dezaparvovec, a one-time gene therapy for the treatment of appropriate adults with the disease.  The therapy […]

The latest episode of the DDW Highlights podcast is now available to listen to below. DDW’s Megan Thomas narrates five key stories of the week to keep DDW subscribers up-to-date on the latest industry news.  Gene therapies have made the headlines over the last week, with treatments for rare AADC deficiency, lung cancer and haemophilia B given […]

News round-up for 21-25 November by DDW Digital Content Editor Diana Spencer  Gene therapies have made the headlines over the last few days, with treatments for rare AADC deficiency, lung cancer and haemophilia B given the go ahead from regulators. This week has also seen the first immunomodulatory treatment for type 1 diabetes approved in the […]

The US Food and Drug Administration (FDA) has approved Hemgenix (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy for appropriate adults with haemophilia B.   The drug is approved for the treatment of adults with haemophilia B who currently use factor IX prophylaxis therapy, or have current or historical life-threatening haemorrhage or have repeated, serious […]

DDW Editor Reece Armstrong speaks to Stephanie Seremetis, Chief Medical Officer, Rare Blood Disorders at Novo Nordisk to get her response to the company’s latest study results for its Mim8 antibody. Earlier this year, pharmaceutical company Novo Nordisk announced positive results from Phase I & II clinical trials investigating its antibody treatment for haemophilia A.   […]

Novo Nordisk has announced positive interim results for its Mim8 antibody which is being as a treatment for haemophilia A.   Novo Nordisk presented interim results from the Phase I & II FRONTIER1 dose-escalation study at International Society of Thrombosis and Haemostasis Annual Congress (ISTH 2022) in London, UK.    The study is assessing the safety and […]