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Cinryze Prophylaxis Approval Leaves Window For HAE Treatment Contenders

Executive Summary

The approval of ViroPharma/Lev's hereditary angioedema therapyCinryze on Oct. 10, with orphan status for prophylaxis only, leaves the window open for other C1 esterase inhibitors to claim the prize of orphan exclusivity for treatment of acute attacks. But ViroPharma thinks it can win that too

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With four drugs in the space, hereditary angioedema is an unusually well-served orphan disease. But the rare condition still has no clear path for efficacy assessment.

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FDA's approval of CSL Behring's Berinert for treatment of acute attacks of hereditary angioedema specifically mentions only two types of attacks. By approving the C1 esterase inhibitor with a targeted indication, the agency could be leaving open a window in the orphan exclusivity for another approval

Berinert Claims Another Part Of The HAE Orphan Market, But May Leave Room

FDA's approval of CSL Behring's Berinert for treatment of acute attacks of hereditary angioedema specifically mentions only two types of attacks. By approving the C1 esterase inhibitor with a targeted indication, the agency could be leaving open a window in the orphan exclusivity for another approval

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