Therapy Trends KOL Insight: Haemophilia A [2019]

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Publication Date:
May 2019
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Are novel drugs set to revolutionise Haemophilia A? 

Predominantly the treatment of choice for Haemophilia A (HA) patients is recombinant FVIII (rFVIII) therapy. Though the majority of HA patients can be managed by replacing FVIII, the development of inhibitors (anti-drug antibodies) to the administered factor continues to be a serious complication. The availability of emicizumab (Hemlibra; Roche) has provided a much needed breakthrough for patients but effective treatment continues to be challenging. Novel factor and non-factor modalities including gene therapy are emerging through the pipeline, but which candidates will stand out? Want to find out more?

Learn how KOLs see the HA market evolving in Therapy Trends KOL Insight: Haemophilia A. Twelve US and European KOLs provide candid insightson 14marketed and 14 pipeline therapies for the treatment of HA.

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Top takeaways

  • How do KOLs rate the current treatment options for HA? What are the major challenges and how effective are the current modalities?
  • Genentech/Roche’s Hemlibra, a bispecific antibody with concomitant binding to factors IXa/X, is considered a breakthrough for patients with inhibitors. Do experts see a role for the mAb in the non-inhibitor segment?
  • How do KOLs rate the newer long-acting non-PEGylated rFVIII therapies in HA? Can CSL Behring’s Afstyla and Bioverativ’s Elocta/Eloctate play an effective role?
  • Can newer PEGylated products make an impact in HA? In a crowded market,Bayer’s Jivi Novo Nordisk’s Esperoct and Takeda’s Adynovate/Adynovi are vying for share but how do KOLs assess their chances?
  • Will standard half-life (SHL) rFVIII concentrates continue to have a role? With the introduction of EHLs, will Novo Nordisk’s NovoSeven RT and Takeda’s FEIBA become obsolete?
  • How do KOLs expect the emergent FVIII approaches to fare in HA? Can Amunix/Bioverativ [Sanofi]’s BIVV 001, Kaifeng’s FRSW 107 and Ascension’s SelectAte play an effective role?
  • Is there an opportunity for rFVIIa therapies? rEVO Biologics/LFB’s  LR 769 and OPKO Health’s MOD-5014 are in Phase III and II, respectively, but how do KOLs rate their prospects?
  • How do experts view the potential of gene therapy? With three Phase III programmes (BioMarin’s valoctocogene roxaparvovec [BMN270], Sangamo/Pfizer’s PF07055480 and Spark Therapeutics’ SPK 8011/SPK 8016) under way, what products standout and why?
  • Will anti-TFPI (tissue factor pathway inhibitor) mAbs have a role in HA? Novo Nordisk’s concizumab and Bayer’s BAY 1093884 are in Phase II but how do KOLs gage their future?
  • Will anti-TFPI (tissue factor pathway inhibitor) mAbs have a role in HA? Novo Nordisk’s concizumab and Bayer’s BAY 1093884 are in Phase II but how do KOLs gage their future?
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“One clear unmet need is the problem of inhibitor development. Another problem is the frequent need for intravenous infusions. Also, there is still the problem of joint disease, even in patients on prophylaxis; it's delayed, but treatment is still not perfect.”
US Key Opinion Leader

“New treatments are coming along which are delivering much better, consistent levels of haemostatic potential, but we still need an improvement in care for patients. That remains an unmet need.” 
US Key Opinion Leader

Sample of therapies covered

Marketed Therapies

  • Advate (Takeda)
  • Adynovate/Adynovi (Takeda)
  • Afstyla (CSL Behring)
  • AryoSeven (AryoGen Pharmed)
  • Elocta/Eloctate (Bioverativ/Swedish Orphan Biovitrum)
  • Esperoct (Novo Nordisk)
  • FEIBA (Takeda)
  • Hemlibra (Roche)
  • Jivi (Bayer HealthCare)
  • Kogenate (Bayer)
  • NovoEight (Novo Nordisk)
  • NovoSeven RT (Novo Nordisk)
  • Nuwiq (Octapharma)
  • Xyntha (Pfizer)

Pipeline Therapies

  • BAY 1093884 (Bayer HealthCare)
  • BIVV 001 (Amunix/Bioverativ [Sanofi])
  • Concizumab (Novo Nordisk)
  • DTX 201 (Bayer HealthCare/Dimension Therapeutics)
  • Fitusiran (Alnylam/Sanofi)
  • FRSW 107 (rFVIII-Fc fusion protein; Kaifeng Pharmaceutical)
  • LR 769 (rFVIIa; rEVO Biologics/LFB)
  • MOD 5014 (FVIIa-CTP; OPKO Biologics)
  • PF 07055480 (SB525; Sangamo Therapeutics/Pfizer)
  • SelectAte (TheraPEG FVIII; Ascension Healthcare)
  • SPK 8011 (Spark Therapeutics)
  • SPK 8016 (Spark Therapeutics)
  • TAK 754 (SHP-654; Takeda)
  • Valoctocogene roxaparvovec (BMN270; BioMarin Pharmaceutical)

KOLs interviewed

KOLs from North America

  • Christopher Walsh, MD, PhD, Professor of Haemostasis and Thrombosis, Division of Hematology and Medical Oncology, Mount Sinai Medical Center, New York, NY
  • Cindy Lessinger, MD, Professor of Medicine, Tulane University School of Medicine, New Orleans, LA
  • Craig Kessler, MD, Professor of Medicine and Pathology, Director of the Comprehensive Hemophilia and Thrombophilia Treatment Center, Georgetown University Medical Center, Washington, DC
  • Michael Callaghan, MD, Professor of Haemostasis and Thrombosis, Division of Pediatric Hematology/Oncology, Carmen and Ann Adams Department of Pediatrics, Children's Hospital of Michigan, Detroit, MI
  • Miguel Escobar MD, Associate Professor, Hematology, McGovern Medical School, The University of Texas Health Science Center, Houston, TX
  • Mindy Simpson, MD, Department of Pediatrics, Hemophilia and Thrombophilia Center, Rush University Medical Center, Chicago, IL

KOLs from Europe

  • Anonymous German KOL, Prof., Professor of Haematology and Director of the haemophilia treatment centre at a teaching hospital, Germany
  • Anonymous Swedish KOL, MD, PhD, Professor of Medicine, Director of Haematology at a teaching hospital, Sweden
  • Claude Negier, MD, Professor of Haemostasis and Thrombosis, Hôpital Louis Pradel, University Claude Bernard Lyon 1, Bron, France
  • John Pasi, MB ChB, PhD FRCP FRCPath FRCPCH, Professor of Haemostasis and Thrombosis, Barts and The London School of Medicine and Dentistry, Queen Mary, University of London, UK
  • Massimo Morfinfi, MD, member of the Medical Advisory Board of the World Federation of Hemophilia (WFH) and former President of Italian Association of Haemophilia Centres (AICE), Via Pace 9, Milan, Italy
  • Rafael Parra Lopez, MD, PhD, Unidad de Hemofilia Hospital Universitari Vall d'Hebron Paseo Vall d'Hebron, Barcelona, Spain

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