BURLINGTON, Mass., Dec. 18, 2015 /PRNewswire/ -- Decision Resources Group finds that the anticipated approvals of several prophylactic agents, including BioCryst's avoralstat and BCX-7353, Dyax's DX-2930, and CSL Behring's CSL-830, will usher in a new era in the management of hereditary angioedema (HAE), marked by a fundamental shift away from treatment of acute episodes towards extensive use of routine prophylaxis. Whereas efficacy, tolerability, or delivery limitations restrict the current use of tranexamic acid, androgens, or C1-inhibitors—Shire's Cinryze and CSL Behring's Berinert—as prophylactics in HAE, the emerging agents are efficacious, safe, and available in oral or subcutaneous formulations, greatly improving patient convenience. Our research, covering the United States and EU5 markets, reveals that HAE patients who are currently on long-term prophylaxis are expected to switch to the novel prophylactic agents. Further, patients who currently forgo prophylaxis and instead opt to treat frequent acute swelling episodes as they occur are expected to initiate long-term prophylaxis with emerging agents to manage their disease.
Other key findings from the Niche Markets and Rare Diseases report entitled "Hereditary Angioedema":
- Owing to the rarity of the disease, a large portion of HAE patients either experience delayed diagnosis or remain undiagnosed, primarily due to the lack of familiarity of physicians to whom patients typically present, including primary care and emergency department physicians, as well as gastroenterologists. We anticipate that the number of diagnosed prevalent cases of HAE will slightly increase over the ten-year study period.
- HAE is associated with high disease burden and poor quality of life. Acute swelling attacks can be painful and even life-threatening, while the fear of an impending attack, or the need for burdensome prophylactic treatment, commonly results in depression and anxiety.
- Multiple effective, safe, and well tolerated therapies are available to manage acute HAE attacks. Timely access to these agents has greatly reduced emergency room visits and hospitalizations among HAE patients. CSL-Behring's Berinert, Shire's Cinryze and Firazyr, Dyax's Kalbitor and Pharming/Valeant's Ruconest are all efficacious on-demand therapies. According to interviewed experts, prescribing of current therapies is largely driven by patient preference.
Comments from Decision Resources Group Analyst Jing Wu, M.S., M.B.A.:
- "HAE is caused by low levels of functional C1-inhibitor, and plasma derived C1-inhibitor replacement drugs Cinryze and Berinert are efficacious and safe prophylactic agents. However, both agents require twice-weekly intravenous administration, which poses a substantial treatment burden for patients who need chronic prophylaxis."
- "As more HAE patients with frequent acute attacks are expected to switch to routine prophylaxis once new agents with convenient delivery become available, the need for on-demand therapies will decrease dramatically. However, we expect rapid uptake of novel routine prophylactic options to offset the anticipated reduction in the use of on-demand therapies and become the key driver of the forecast expansion of the HAE therapeutic market. Owing to its strong clinical efficacy and convenient biweekly subcutaneous dosing, Dyax's DX-2930 in particular will be well positioned for uptake as a routine HAE prophylactic agent following launch."
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SOURCE Decision Resources Group