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Second C1 Inhibitor Treatment Approved for HAE

11 Oct

The FDA released this statement on October 9th:

The U.S. Food and Drug Administration today approved Berinert, the first treatment for acute abdominal attacks and facial swelling associated with a rare and potentially life-threatening genetic disease called hereditary angioedema (HAE).

Berinert is approved for adults and adolescents with HAE, which can occur spontaneously or during stress, surgery, or infection in patients diagnosed with HAE. The symptoms during abdominal attacks include severe abdominal pain, nausea, vomiting, cramps, and diarrhea.

“Berinert will enhance the treatment options for individuals who experience acute abdominal attacks and facial swelling associated with hereditary angioedema,” said Karen Midthun, M.D., acting director of FDA’s Center for Biologics Evaluation and Research.

Berinert is a protein product derived from human plasma. It regulates clotting and inflammatory reactions that, when impaired, can lead to local tissue swelling. In a clinical trial of 124 adults and adolescents with C1 esterase, inhibitor deficiency, Berinert was shown to be effective at treating the symptoms of acute moderate to severe abdominal attacks and facial swelling in patients with HAE.

Berinert is contraindicated in patients with a history of life-threatening hypersensitivity reaction to C1 esterase inhibitor preparations.  The most serious adverse reaction reported in clinical studies was an increase in the severity of pain associated with HAE. The most common adverse reactions include subsequent HAE attack, headache, abdominal pain, nausea, muscle spasms, pain, diarrhea and vomiting.

Berinert is manufactured by CSL Behring, Inc., Marburg, Germany.

This announcement has two implications:

First, there are now two C1 inhibitor products derived from human blood available in the US.  Both are intravenous infusions:  Cinryze and Berinert.  Both can/could be used preventatively (Berinert off-label) and acutely (Cinryze off-label).

The second implication is that because Berinert received FDA approval for acute attacks, i.e., attacks already in progress, CSL Behring has the sole right to market their product for that purpose for the next seven years under the Orphan Drug Act.  Even though Cinryze works perfectly well for acute attacks, ViroPharma is not permitted to state this use in its prescription literature (but doctors can still use Cinryze off-label for acute attacks).  ViroPharma has and did submit data supporting the acute use of Cinryze, but I think it was in the US patient community’s best interest for the FDA to approve Berinert for acute use instead because such a move now makes two products available (and hence competition, eventually) in the US. This is especially important for patients who have not responded or are experiencing a decreased response to Cinryze.

Conversely, Berinert could be used preventatively, off-label, but I believe it has a shorter half-life than Cinryze, which makes its use as a prophylactic impractical — for instance, if I were to switch from Cinryze to Berinert for prophylactic treatment, I’d be going from infusions every other day to (possibly) twice a day.  However, I am not 100% certain about the exact effects of the half-life, so don’t take this as gospel.  I’m smart, but I’m no scientist and (thank the Lady) and I ain’t no doctor.

Thus far, I’d have to say it’s been a wonderful new century for sufferers of HAE – hooray!!


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