European regulators have approved a label extension for Shire’s Firazyr, broadening its use include adolescents and children aged two years and older with a particular type of HAE.
Firazyr (icatibant) was approved in 2008 for adults with C1-esterase-inhibitor (C1-INH) deficiency, expanding its reach to children offers a new treatment option for relief of symptoms.
HAE is a rare genetic disease characterised by recurrent attacks of localised oedema, most commonly affecting the extremities, gastrointestinal tract and, less frequently, upper airways.
“As a long-term partner to the HAE community, we understand the unique burden this disease places on children living with HAE and their caregivers,” said Jennifer Schranz, Global Development lead, HAE, Shire.
“This approval in Europe demonstrates our unwavering commitment to helping patients and represents a significant advance for paediatric patients, who now have a subcutaneous treatment option for acute HAE attacks.”
Approval was based on an open label, non-randomised, single-arm study, with a primary efficacy endpoint of time to onset of symptom relief (TOSR). Overall, median TOSR was 1.0 hour, with no differences between children and adolescents. More than 70 percent of patients experienced symptom relief at 1.1 hours, and more than 90 percent by 2 hours post-treatment.
The study showed Firazyr was well tolerated and demonstrated rapid resolution of symptoms during an HAE attack through a single injection, Shire noted.