BURLINGTON, Mass., Dec. 1, 2016 /PRNewswire/ -- Decision Resources Group – Hemophilia patients will have a greater variety of treatment options from 2019, with the launch of several novel agents including gene therapies. Hoping to improve the dosing burden that hemophilia A patients currently experience, Roche / Chugai's ACE-910 (emicizumab) has provoked much excitement with its potential to offer once-weekly subcutaneous injections. ACE-910, along with CSL Behring's CSL-689, will also address a major unmet need in hemophilia and may make prophylactic treatment a reality for inhibitor patients. The overall need for novel agents to treat Hemophilia B is relatively low; extended half-life factor IX products provide effective treatment options for this patient group.

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Key findings from the Niche Disease Landscape and Forecast report titled Hemophilia:

  • Hemophilia A is more commercially attractive than hemophilia B. Hemophilia A has a larger patient population and a greater unmet need than hemophilia B, which positively influences R&D and disease visibility. These market characteristics are reflected in a larger and more diverse number of candidates forecast to launch for hemophilia A with or without inhibitors, through 2025.
  • Extended half-life (EHL) factor IX products will retain a strong foothold in the hemophilia B market. In contrast to the EHL factor VIIIs for hemophilia A, EHL factor IX products such as CSL Behring's Idelvion and Biogen / Swedish Orphan Biovitrum's Alprolix have achieved meaningful increases in half-life extension. Physicians interviewed by DRG specifically refer to the longer half-life making a significant impact in the management of hemophilia B patients. As such, the bar is set high for future product launches to demonstrate improvements.
  • Inhibitor patients will be better served through 2025. Novel products ACE-910 and CSL-689 will make prophylaxis possible for inhibitor patients, and will minimize the clinical impact and physical consequences of bleeds in this patient group.
  • Physicians are expressing concerns over the use of PEGylated products. Physicians have mixed views on the long-term toxicity of intravenously administered PEGylated products. We note particularly negative ill sentiment around the use of PEGylated EHL products in children.
  • Gene therapy and gene editing therapy not likely to offer 100% cure rates. We anticipate a mixed picture for both gene therapy and gene editing products in addressing the clinical implications of the troughs associated with regular factor IX or factor VIII replacement. Interviewed physicians also report little interest in gene therapy from parents of children or adolescents with hemophilia.
  • Dosing is not always predictable with current treatments, and emerging agents will not fully address this challenge. Treatment protocols and their associated dose ranges can be highly variable both between and within patients. The heterogeneity of the hemophilia population decreases dosing predictability and can drive up consumption and cost.

Comments from DRG's Business Insights Analyst, Kerri Brown, M.Pharm.:

  • "The hemophilia A landscape will start to look very different from 2019, with the launch of some innovative agents like Roche / Chugai's ACE-910 and Alnylam's fitusiran and following on from these, the hemophilia A gene therapies. These new agents have an opportunity to make a big impact on the adherence rate and clinical outcomes achievable for severe hemophilia A patients, who are still largely undertreated today.
  • "The extended half-life factor IX products are here to stay, and they will make it difficult for new hemophilia B products to gain market share"
  • "Effective treatment options for the hemophilia inhibitor population are eagerly awaited. We expect ACE-910 and CSL-689 to go a long way to address this large unmet need, and will make prophylactic treatment a reality for inhibitor patients."

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About Decision Resources Group
DRG, a subsidiary of Piramal Enterprises Ltd., offers best-in-class, high-value data, analytics and insights products and services to the healthcare industry, delivered by more than 1000 employees across 17 offices in North America, Europe and Asia. DRG provides the Life Sciences, Provider, Payer and Financial Services industries the data, tools, insights and advice they need to compete and thrive in an increasingly complex and value-based marketplace. decisionresourcesgroup.com.

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