European Approval for Label Extension of Firazyr

The European Commission (EC) has approved a label extension granting a new indication for Firazyr (icatibant injection), broadening its use to adolescents and children aged 2 years and older, with HAE caused by C1-esterase-inhibitor (C1-INH) deficiency. Firazyr has been approved in the European Union (EU) since 2008 for symptomatic treatment of acute attacks of HAE in adults with C1-INH deficiency.

“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 plc. “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.”

The use of Firazyr in paediatric patients was studied in an open label, non-randomised single-arm study, involving 32 paediatric patients with HAE. The efficacy population consisted of 11 children and 11 adolescents with attacks. The primary efficacy endpoint was time to onset of symptom relief (TOSR) based on the investigator-assessed composite post-treatment symptom score, defined as earliest time post-treatment when 20% or more improvement in the composite symptom score was achieved, without worsening of any single component score.

Overall, median TOSR was 1.0 hour, with no differences between children and adolescents. More than 70% of patients experienced symptom relief at 1.1 hours, and more than 90% by 2 hours post-treatment.

The majority of paediatric patients who were treated with subcutaneous Firazyr experienced injection site reactions such as erythema, swelling, burning sensation, skin pain and itching/pruritus; these were found to be mild to moderate in severity and consistent with reactions that have been reported in adults.

The study showed Firazyr was well tolerated and demonstrated rapid resolution of symptoms during an HAE attack through a single injection. The study that led to this approval is the first and only trial investigating a subcutaneous therapy in the HAE paediatric population.

“Due to the unpredictable and debilitating nature of HAE attacks, children living with the condition can benefit from having a new treatment option that can provide symptomatic relief of acute HAE attacks with a subcutaneous injection,” said Henrik Balle Boysen, Executive Director of HAEi. “Clinical work to bring treatment options to younger patients is vitally important and greatly appreciated by the global HAE community.”

Firazyr will be available for use in paediatric patients in Europe beginning in Q4.
(Source: Shire)

2017-10-26T23:38:12+00:00