CSL Behring has submitted the BLA for its hereditary angioedema therapy C1-INH, a C1-esterase inhibitor concentrate, the company said March 6. The drug is marketed in Europe as Berinert .

The BLA is based on a recently completed Phase II/III trial, IMPACT, that enrolled 124 patients with acute, moderate or severe abdominal or facial attacks, King of Prussia, Pa.-based Behring said. The main study endpoints were time to onset of relief and proportion of subjects with worsening clinical symptoms, as well as safety.

HAE is a genetic swelling disorder that can involve painful attacks of the digestive system or affect the face and larynx and result in asphyxiation. There currently is no approved therapy, but at least four firms in additional to Behring have products in development, aiming to be first to market and gain extended patent protections from their orphan disease designation.

Behring estimates 6,000 to 10,000 people suffer from the disease in the U.S.

In January, New York-based Lev Pharmaceuticals received a "complete response" letter from FDA on the BLA for its C1-esterase inhibitor Cinryze (¹ Also see "FDA “Approvable” For Lev’s Cinryze Opens Window For Jerini To Reach Market First" - Pink Sheet, 31 January, 2008.).

Meanwhile, Berlin-based Jerini has an April 26 action date for its HAE therapy, a bradykinin blocker called Firazyr (icatibant). A scheduled February FDA advisory committee review of Firazyr was cancelled in January (² Also see "Advisory Committee Review Of Firazyr Canceled" - Pink Sheet, 8 January, 2008.). The product failed to meet a primary endpoint on time to onset of symptom relief in one pivotal trial but showed efficacy in another.

Leiden, Netherlands-based Pharming completed a clinical trial of its candidate Rhucin , a recombinant human C-1 inhibitor, in the fourth quarter of 2007 and announced Jan. 29 that it plans to have summary data available in this quarter. A U.S. open-label study is ongoing, and Pharming said it plans to file a BLA this year.

In December, the European Medicines Agency's Committee for Medicinal Products for Human use recommended against approval of Rhucin, but Pharming is appealing the decision and plans to bolster its application with data from the U.S. trial.

Dyax, in Cambridge, Mass., initiated a confirmatory open-label Phase III study of its candidate DX-88 (ecallantide), a recombinant kallikrein inhibitor, in April of last year under a special protocol assessment with FDA.

-Shirley Haley (s.haley@elsevier.com)

CSL Behring Submits C1-INH For Hereditary Angioedema

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