Shire's Cinryze wins expansion for children over six

Shire has won approval from the US drug regulator to expand the offering of its Cinryze treatment to children aged six years and older with hereditary angioedema.

The US Food & Drug Administration had already okayed the C1 esterase inhibitor in 2008 for routine prophylaxis against attacks in adolescents and adults living with the rare, genetic disorder, which results in recurring swelling in various parts of the body.

HAE, which can be life-threatening when attacks result in obstruction of the airways, is estimated to affect between 1 in 10,000 to 1 in 50,000 people worldwide.

In the first quarter of the year, Shire saw sales of Cinryze decline compared to the same time last year due to destocking and the impact on demand from a competitor launch.

Shire's head of research and development, Andreas Busch, said: “Symptoms of HAE often present in childhood with the average child experiencing their first HAE attack around the age of 10. With the FDA label expansion of Cinryze, children as young as six years old living with HAE now have the first FDA approved treatment option available to help prevent attacks.”

The approval, which follows a similar European approval more than a year ago, was based on data from a dedicated clinical study that evaluated the use of the drug in 12 patients living with HAE aged between seven and 11, which found a mean reduction in the number of attacks of between 71.1% and 84.5% depending on the level of dose. Some adverse reactions, including headache, nausea, fever and some redness of the skin around the site of infusion, but none of these adverse reactions were severe or led to discontinuation.

“This news is exciting for the HAE community because those living with HAE who are as young as 6 have a new option to help prevent attacks,” said Anthony Castaldo, president of the US Hereditary Angioedema Association.