GENEVA - Shire PLC won US 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. and Dyax Inc.
Firazyr was approved for acute attacks of hereditary angioedema in people aged 18 years and older, the Food and Drug Administration said in a statement yesterday. The therapy has been approved for use in Europe since 2008.
The medicine is the first treatment for attacks 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 yesterday. “This will give them really good, strong differentiation.’’
Fewer than 30,000 people in the United States have hereditary angioedema, the FDA said in the statement. 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.
“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 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 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.