I attended the WFH Virtual Summit and, although I would obviously have preferred to be there in person, I was impressed by the quality of the speakers’ presentations. There were some minor technical problems but those are inherent in such large events.
I focussed on the medical sessions, especially the one on currently available and upcoming treatments entitled Treatments for bleeding disorders: Current environment and pipeline products.
I was particularly interested in the first speaker, Dr. Flora Peyvandi, professor of internal medicine at the University of Milan, Italy, who said that the treatments for bleeding disorders have progressed dramatically over the past decade. She cited the advent of factor concentrates with extended half-life (FVIII: Adynovate, Eloctate, Esperoct, JIVI; FIX: Alprolix, Idelvion, Rebinyn), single-chain factor VIII concentrates (Afstyla) and bispecific antibodies (Hemlibra). In fact, preliminary results from several ongoing clinical trials around the world found that the use of FVIII concentrates with extended half-life resulted in an average reduction in infusions of 39% and an average reduction in the consumption of FVIII concentrates of 16%. For FIX, the results are even more dramatic with infusions down by 51% and consumption reduced by an average of 39%.
Other products are in clinical trials, and some are so advanced that they could be on the market soon. Dr. Peyvandi discussed other single-chain factor VIII concentrates (BIVV001, SubQ8, FVIIIr with rD’D3-FP), other bispecific antibodies (Mim8, KY1049), anti-TFPI antibodies (for Tissue Factor Pathway Inhibitor) [Concizumab, Marstacimab, BAY-1093884, MG1113], APC inhibitors (for Anaphase Promoting Complex) [SerpinPC], and siNRAs (small interfering RNA) [Fitusiran]. These techniques have the potential to develop products that would substantially increase the activity of FVIII or FIX compared to current extended half-life products for patients with or without inhibitors. Obviously, the real efficacy of these products and their safety still need to be proven once more patients have used them over a longer period of time. But the results are very encouraging.
The final speaker in the session was Dr. Johnny Mahlangu, clinical hematologist at the Charlotte Maxeke Johannesburg Academic Hospital and professor of hematology at the University of the Witwatersrand in Johannesburg, South Africa, who spoke about the opportunities for patients to take part in clinical studies, particularly in emerging countries. This is often a way for patients to access care and treatment in countries where very few products are purchased per capita because of limited resources. According to the statistics he presented, industrialized countries, which account for 24% of the world’s population, consume 77% of the total, in international units, of factor concentrates used in the world. A huge imbalance!
Dr. Mahlangu concluded by saying that, over time, the goals of treatment for bleeding disorders have evolved. The objectives include 1) supportive care, 2) no infection, 3) no bleeding, 4) no inhibitors, and now it is important to add 5) access to effective treatment, no matter what it is or where patients are living.
I would add that this paradigm also applies to industrialized countries where patients have to deal with government authorities who have the power to decide what treatments to offer in a health care system paid for by taxpayers. This is already a problem in some Canadian provinces, and accessibility will be quite a challenge with all the innovative products coming along, including gene therapy. New ways of proceeding will have to be developed and introduced.
Everything you wanted to know about novel treatments … and more
July 21, 2020 – The European Haemophilia Consortium has just released the first issue in 2020 of Novel treatments in haemophilia and other bleeding disorders: A periodic EHC Review.
This periodic review summarizes the latest research on therapies for hemophilia A and B, von Willebrand disease and novel treatments for rare bleeding disorders. – D.P.