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New Hemophilia Therapies on the Horizon

Decision Resources forecasts market innovations to 2025

Decision Resources Group, a health care research and consulting company based in Burlington, Massachusetts, forecasts that several therapies will launch for hemophilia A, hemophilia B, and hemophilia with inhibitors in the U.S. during the 10-year period between 2015 and 2025. New factor IX agents will have a significant affect on the hemophilia B market, but new factor VIII drugs will fail to make a strong impression on the hemophilia A sector. The launch of agents with novel mechanisms of action will provide competition for classic factor-replacement therapies in hemophilia A and hemophilia A with inhibitors. Gene therapy for hemophilia B is forecast to launch late in the study period.

According to the analysts at Decision Resources, extended half-life factor IX products are set to revolutionize the treatment of hemophilia B. The emergence of longer-acting agents, such as Alprolix (Biogen) and CSL654 (CSL Behring), offer significant advantages in dosing intervals (decreased frequency of infusions). By 2025, the company predicts that Alprolix and CSL654 will capture most of the market share among the extended half-life factor IX products.

Despite the expected launch of several new drugs for hemophilia A, the high unmet need for hemophilia A patients will remain. The analysts at Decision Resources do not expect the extended half-life factor VIII concentrates to significantly affect the hemophilia market. This weak performance will be a result of the marginal increase in half-life offered by these products; the frequency of infusions with the longer-acting factor VIII products is similar to the frequency of infusions given with standard half-life products. The analysts forecast that Eloctate (Biogen) and CSL 627 (CSL Behring) will capture most of the market share among the extended half-life factor VIII products. Personalized prophylaxis regimens and pharmacokinetic tailoring are expected to become increasingly popular during the 10-year study period.

Inhibitor patients remain one of the major challenges in hemophilia, particularly severe hemophilia A patients, the company says. Bypass agents are required in 20% to 30% of patients in whom immune tolerance induction is ineffective. Physicians’ concerns regarding the half-life, clinical efficacy, and cost-effectiveness of currently available agents will be addressed by the launch of CSL 689 (CSL Behring), LR769 (rEVO), and ACE910 (Chugai/Roche). ACE910 is a highly anticipated and novel way of prophylactically treating hemophilia A patients with inhibitors, according to the analysts.

The company also predicts that gene therapy will make a significant contribution to the current unmet need in hemophilia A, whereas it may not be necessary in hemophilia B. The longer-acting hemophilia B treatments are largely viewed as safe and will have had a significant affect on clinical outcomes by the time the gene therapy BAX 335 launches toward the end of the company’s forecast window.

“The emergence of the FIX longer-acting factor concentrates [LAFCs] will revolutionize the treatment of hemophilia B, while the FVIII LAFCs will not make a significant impact in the treatment of hemophilia A,” analyst Kerri Brown said. “For this reason, we expect to see an increase in personalized prophylaxis and pharmacokinetic tailoring in severe hemophilia A through 2025.

“Emergence of longer acting agents and transgenically produced agents, for hemophilia with inhibitors, will address physician concerns about the half-life of current bypass agents and cost of therapy, respectively,” Brown added. “Launch of the novel agent ACE910 toward the tail end of our forecast window has the potential to revolutionize the treatment of hemophilia A and hemophilia A with inhibitors.”

Source: PR Newswire; December 28, 2015.

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Decision supported by data from more than 4,000 patients