Shire Plc (SHP) won U.S. marketing approval for Firazyr, the first self-injectable drug to treat attacks of a rare genetic swelling disease, giving it an edge over products by CSL Ltd. (CSL) and Dyax Inc.
Firazyr was approved for acute attacks of hereditary angioedema in people aged 18 years and older, the U.S. Food and Drug Administration said in a statement today. The therapy has been approved for use in Europe since 2008, and permitted as a self-administered treatment since March.
The medicine is the first treatment for attacks of HAE that patients can inject themselves, providing an advantage over CSL’s Berinert and Dyax’s Kalbitor, both of which must be given by a doctor. The treatment may generate $48 million in sales next year and $199 million by 2017, according to analyst estimates compiled by Bloomberg.
“They will get market share,” Navid Malik, an analyst at Matrix Corporate Capital LLP in London, said in a telephone interview today. “This will give them really good, strong differentiation.”
Shire fell 57 pence, or 2.8 percent, to 1,955 pence in London trading, after earlier declining as much as 5.4 percent in the session. The company said late yesterday that it was scrapping development of its Dermagraft skin substitute for leg ulcers after the treatment failed in a trial.
Fewer than 30,000 people in the U.S. have hereditary angioedema, the FDA said in the statement. HAE patients have low levels or improper functioning of a protein called C1 inhibitor, causing potentially fatal attacks of inflammation in the limbs, face, intestinal tract and windpipe.
‘Important New Option’
“The unpredictability of HAE attacks can place limits on patients’ travel, employment opportunities, educational opportunities, and social life,” Janet Long, executive vice president of the U.S. HAE Association, said in a statement distributed by Shire. “With this approval, patients have an important new option to treat HAE attacks.”
In three trials, patients who injected themselves with Firazyr got relief from the symptoms of HAE attacks in a median of two hours, compared with almost 20 hours in patients who received a placebo, the FDA said in the statement. The most common side effects were reactions at the injection site, fever, increased liver enzymes, dizziness and rash.
A panel of advisers to the FDA voted 12-1 in June in favor of approving the treatment, and 11-1 in favor of allowing patients to self-administer it, with one abstention.
Firazyr, also known as icatibant, generated revenue of $11 million last year after being introduced in Europe in 2008. Including the U.S., it has now been approved in 38 countries, Shire said in the statement.
CSL, based in Melbourne, won FDA approval for Berinert in October 2009 to treat facial and abdominal attacks of the disease. Two months later, the U.S. regulator cleared Kalbitor from Cambridge, Massachusetts-based Dyax, for acute attacks in patients aged 16 or older.
ViroPharma Inc. (VPHM), of Exton, Pennsylvania, sells a medicine called Cinryze, which patients self-administer every three to four days to prevent attacks of HAE. A fifth drug for the disease is under development by Leiden, Netherlands-based Pharming Group NV (PHARM) and Santarus Inc. of San Diego.
Shire gained Firazyr with its acquisition of Jerini AG for 349 million euros ($501 million) in September 2009. The FDA rejected Firazyr in April 2008, citing questions about its efficacy.
The drug is the latest from Shire’s genetic-therapies division, which the company is betting on to reduce reliance on older treatments such as its attention-deficit disorder pill Adderall XR.
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