Monitoring International Trends posted January 2015

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Monitoring International Trends

posted January 2015

The NBA monitors international developments that may influence the management of blood and blood products in Australia. Our focus is on:

  • Potential new product developments and applications;

  • Global regulatory and blood practice trends;

  • Events that may have an impact on global supply, demand and pricing, such as changes in company structure, capacity, organisation and ownership; and

  • Other emerging risks that could potentially put financial or other pressures on the Australian sector.

A selection of recent matters of interest appears below. Highlights include:

  • The submission of biological licence applications to the US Food and Drug Administration (FDA) for:

    1. Bayer’s BAY 81-8973 for haemophilia A in adults and children

    2. CSL Behring’s long-acting fusion protein linking recombinant factor !X with albumin

    3. Baxter’s extended half-life recombinant factor VIII, BAX 855, and its treatment for von Willebrand disease BAX 111

  • Approval by the FDA of Cerus Corporation’s Intercept Blood System for platelets and its Intercept Blood System for plasma

  • Publication in the January issue of Anesthesiology of studies of studies on two specific risks posed by blood transfusions: transfusion-related acute lung injury and transfusion-associated circulatory overload. These are the two most significant causes of post-transfusion mortality in the US.

  • The curing of haemophilia in mice models using a new genome editing method

  • The US review of platelet transfusions which suggested that they were associated with increased odds of dying in the hospital, fivefold for patients with heparin-induced thrombocytopenia and double for patients with thrombotic thrombocytopenic purpura.


1.Products 1

Haemophilia treatments 1

Sickle Cell Disease (SCD) 2

Other 3

Market size 4

 Research and Markets: Global Plasma Fractionation (Albumin, Immunoglobulin, Factor VIII, Protease Inhibitors) Market Report 2014-2019 5

2.Regulatory 5

Plasma and recombinant products 5

Blood donation, processing, storage and use; blood substitutes 6

Other 7

3.Market structure and company news 7

4.Country-specific events 10

5.Safety and patient blood management 10

Appropriate transfusion 10

Other. 12

6.Research 13

 Chen Liu, Daniel Kim-Shapiro et al.,“Mechanisms of Human Erythrocytic Bioactivation of Nitrite”, Journal of Biological Chemistry, 12/14 DOI: 10.1074/jbc.M114.609222 14

7.Legal actions and enquiries 14

8.Infectious diseases 15

Mosquito-borne diseases: dengue, chikungunya and malaria 15

Ebola Virus Disease 16

Influenza: strains, spread, prevention and treatment 17


Other diseases: occurrence, prevention and treatment 19

 Hervé Cassard et al., “Evidence for zoonotic potential of ovine scrapie prions”, Nature Communications Article number:5821 Published 16 December 2014 doi:10.1038/ncomms6821 19

  1. Products

Here the NBA follows the progress in research and clinical trials that may within a reasonable timeframe make new products available, or may lead to new uses or changes in use for existing products.

Haemophilia treatments

    1. Alnylam says it is advancing ALN-AT3, an investigational RNAi therapeutic in development for haemophilia and rare bleeding disorders, after positive initial Phase 1 data were recently reported.

    2. LFB announced in late December the achievement of patient enrollment target for PERSEPT 1, a multinational Phase III clinical trial of LR769, a novel recombinant form of human Factor VIIa, in adolescent and adult congenital haemophilia A or B patients with inhibitors. This Phase III trial study is being sponsored by LFB.’s US subsidiary1. Initial results, expected in the first quarter of 2015, will provide the basis for a second Phase III study, PERSEPT 2, which will assess the pharmacokinetics, safety and efficacy of LR769 for the treatment of bleeding episodes in paediatric haemophilia patients with inhibitors. A third study, PERSEPT 3, will evaluate the safety and efficacy of LR769 for prevention of bleeding in patients undergoing surgery. Both these studies are expected to begin in mid-2015.

Sickle Cell Disease (SCD)

    1. At the 56th Annual Meeting of the American Society of Haematology (ASH) in San Francisco, one poster session examined patient and caregiver perspectives on adherence to iron chelation therapy (ICT), which is used to manage iron overload in patients who have repeat transfusions2. A second study provided evidence that blood transfusion improves health-related quality of life for children with SCD3.

    2. The ASH meeting also saw a poster presentation4 on an innovative biochip, which evaluates the biophysical properties of red blood cells in sickle cell patients, and has the potential to become a standard test for monitoring the disease because of its widespread applicability and its use of only small volumes of blood.

    3. Global Blood Therapeutics announced mid-January that the first cohort of eight subjects had been dosed in its Phase I/II clinical trial of GBT440, for the treatment of sickle cell disease. GBT440 is an oral, once daily dosing, direct-acting sickle haemoglobin modifier for the chronic, prophylactic treatment of SCD. The drug works by increasing haemoglobin's affinity for oxygen. Oxygenated haemoglobin does not polymerize, so sickling of red blood cells is blocked.

    4. A tiny microfluidic device to help predict vaso-occlusive crises in SCD has been developed by Ming Dao (Massachusetts Institute of Technology) and colleagues5.


    1. At the ASH meeting, Alexion presented findings for its drug Soliris6 (eculizumab) in the treatment of atypical haemolytic uremic syndrome (aHUS), as well as new data regarding medical care for patients with aHUS and paroxysmal nocturnal haemoglobinuria (PNH)7. Dr. Spero R. Cataland of Ohio State University Medical Center presented the results of two post-hoc sub-analyses from two trials investigating Soliris’ safety and efficacy in children and adult patients with aHUS with or without identified genetic mutations at baseline. Results show that platelet count normalization was achieved by 100 per cent in both paediatric and adult patients with an identified mutation. Platelet count normalization was met by 91 per cent of paediatric and 95 per cent of adult patients without an identified mutation. “Given the life-threatening nature of aHUS and the well-established clinical efficacy of Soliris, these data provide additional evidence for initiating treatment with Soliris immediately upon clinical diagnosis of aHUS. This is particularly important since genetic testing can take several months to complete and, to date, genetic complement mutations can only be identified in 50 per cent to 70 per cent of patients with aHUS,” said Dr. Cataland. Leonard Bell, Chairman and CEO of Alexion, said: “We are pleased that the data presented at ASH continue to expand our understanding of aHUS and PNH… Importantly, significant improvements in haematologic and renal outcomes were observed in paediatric and adult patients with aHUS, both with and without identified genetic mutations, supporting the early initiation of Soliris treatment regardless of mutation status.”

    2. Kamada reported that preliminary results from a Phase I/II clinical study of its human Alpha-1 Antitrypsin (AAT) indicated that continuous administration of AAT as therapy for steroid resistant gut graft-versus-host-disease, or GvHD, is feasible. The Fred Hutchinson Cancer Research Center in Seattle, Washington has been conducting the study in cooperation with Baxter and Kamada. The study is an open label, dose escalation, safety and efficacy study evaluating 24 GvHD patients who suffer from inadequate response to steroid treatment. The primary outcome of the study is to evaluate the efficacy of AAT in ameliorating the severe intestinal inflammation associated with GvHD.

    3. Kamada expects to report results in the first half of 2015 from its US Phase II/III clinical trial its anti-rabies immunoglobulin as a post-exposure prophylaxis. It hopes to file a biologics license application with the FDA before the end of 2015. Kamada has a strategic agreement with Kedrion for the clinical development and marketing of the product in the US.

    4. One in four Jews is a carrier of one or more of the nineteen known preventable Jewish genetic diseases, according to the Center for Jewish Genetics. Although Sephardic Jews and non-Jews can carry these diseases, they appear twice as often for Ashkenazi Jews as they do for the rest of the population. JScreen, launched through the Emory University School of Medicine’s Department of Human Genetics, is an at-home, carrier-screening program.

    5. Pharming Group and Salix Pharmaceuticals announced in January that the first patient had been treated in their Phase II clinical study of Ruconest, (C1 Esterase Inhibitor [Recombinant]) 50 IU/kg, for prophylaxis in patients with hereditary angioedema (HAE). Patients being enrolled are deficient in C1 inhibitor and have a history of at least four attacks per month. The trial is a randomized, double-blind study, in which 30 patients will receive Ruconest either once or twice weekly, or placebo in each of three treatment periods. With the crossover design, all patients will receive each of the dosing regimens. The study is being conducted at sites in Canada, Europe, Israel, and the US.

    6. Cerus announced in December that that its Phase II study on red blood cells when treated with the INTERCEPT blood system has met its primary endpoint. The randomized, single-blind, controlled, multi-center was conducted in 26 healthy subjects. Each subject was given two transfusions of the subject's own red blood cells: one INTERCEPT-treated and the other a control not treated for pathogen inactivation. Preliminary analysis showed that more than 75 per cent of treated red blood cells continued to circulate 24 hours following transfusion, thereby meeting the primary endpoint of the study. The INTERCEPT treated red blood cells had a recovery of 83 per cent compared with 85 per cent for control red blood cells. Both INTERCEPT-treated and control red blood cells met the criteria for red blood cell recovery as recommended by the FDA. Cerus will move the INTERCEPT red blood cell program into Phase III in the U.S. Cerus has already completed a Phase III study on the INTERCEPT red blood cell system in Europe in patients with acute anaemia and is filing for CE mark approval.

    7. Researchers at Héma-Québec produced eye drops using a concentrate of plasminogen to save the sight of a young patient with ligneous conjunctivitis, a rare disorder that occurs in patients with plasminogen deficiency.

Market size

    1. Allied Market Research forecasts the Global Erythropoietin (EPO) market will reach $US 11.9 billion annually by 20208. Europe led the global EPO drug market in 2013 closely followed by North America. Darbepoetin alfa is the fastest growing drug class because of its high potency and minimal side-effects. The market is seeing biosimilars for the 'off-patent' drug erythropoietin alfa9. Rising incidences of cancer, end stage renal disease and HIV are largely responsible for the growing demand for EPO drugs. The kidney therapeutic segment is expected to be the largest by 2020. Companies are investing in research and development to expand product use to newer disorders such as neural diseases and in wound healing.

    2. The global plasma fractionation market is estimated at $US 16,573.4 million for 2014 and growing at a compound annual growth rate of 8.9 per cent during the period of 2014 to 201910. The market is driven by aging population, increasing numbers of haemophilia patients, improved diagnosis, increasing emphasis on prophylaxis, increasing off-label use of albumin and increasing use of mmunoglobulin in chronic diseases.

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