Shire Drug for Rare Swelling Disease Faces Scrutiny by FDA

Shire Plc (SHP)’s experimental treatment for a genetic swelling disease may offer a more convenient option for patients, according to U.S. regulators weighing whether to clear the competitor to CSL Ltd. (CSL)’s Berinert and Dyax Inc. (DYAX)’s Kalbitor.

Self-administered injections of Shire’s icatibant eased symptoms of people with hereditary angioedema in studies reviewed in a Food and Drug Administration staff report released today. Outside advisers to the agency will discuss the findings and make recommendations on approval at a June 23 public meeting in Silver Spring, Maryland.

Hereditary angioedema, or HAE, is estimated to affect one of every 50,000 people, according to the National Institutes of Health. Patients have low levels or improper functioning of a protein called C1 inhibitor, causing attacks of inflammation in the limbs, face, intestinal tract and windpipe. Icatibant, marketed in Europe as Firazyr, would be the first treatment that patients can take by themselves without a doctor.

“The majority of patients reported ease and a preference for self-administration,” FDA staff said in the report. “The frequency and nature of the reported adverse events, including local injection site reactions, were similar to those observed for the injections administered by a health-care professional.”

Pregnancy Concern

Studies in dogs and rabbits raise concerns about potential dangers for pregnant women, the FDA said. While safety data in people haven’t confirmed this risk, the agency is interested in a clinical trial examining icatibant’s effect on reproductive hormones.

American depositary receipts of Dublin-based Shire, each representing three ordinary shares, rose 43 cents to $90.08 at 4 p.m. New York time in Nasdaq Stock Market trading.

Firazyr had revenue of $11 million last year after being introduced in Europe in 2008. The average estimate of five analysts surveyed by Bloomberg calls for sales of the medicine to reach $120 million in 2015.

CSL, of Melbourne, won FDA approval for Berinert in October 2009. Two months later, the agency cleared Kalbitor from Cambridge, Massachusetts-based Dyax. ViroPharma Inc., of Exton, Pennsylvania, sells a medicine called Cinryze to prevent attacks of HAE. A fifth drug for the disease is under development by Pharming Group NV (PHARM) of Leiden, the Netherlands, and Santarus Inc. (SNTS) of San Diego.

To contact the reporters on this story: Catherine Larkin in Washington at clarkin4@bloomberg.net; Anna Edney in Washington at aedney@bloomberg.net

To contact the editor responsible for this story: Adriel Bettelheim at abettelheim@bloomberg.net;

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