EC approves paediatric label extension for Shire's FIRAZYR
Rare disease specialist Shire announced on Thursday that the European Commission has approved a label extension, granting a new indication for FIRAZYR (icatibant injection), broadening its use to adolescents and children aged two years and older with hereditary angioedema (HAE) caused by C1-esterase-inhibitor (C1-INH) deficiency.
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The FTSE 100 company said FIRAZYR had already been approved in the European Union since 2008 for the symptomatic treatment of acute attacks of HAE in adults with C1-INH deficiency.
It described HAE as a “rare genetic disease” characterised by recurrent attacks of localised oedema.
The areas of the body most commonly affected are the extremities, gastrointestinal tract and - less frequently - it could cause life-threatening attacks due to obstruction in the upper airways.
Symptoms of HAE often presented in childhood, Shire explained in its statement, and while attacks could occur at any age, early onset may predict a “more severe” disease course.
"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 Shire’s global development lead for HAE, Jennifer Schranz.
“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.
Shire said 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 the earliest time post-treatment when a 20%-or-more improvement in the composite symptom score was achieved, without worsening of any single component score.
Overall, median TOSR was one hour, with no differences between children and adolescents.
Shire said 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,” Shire’s board said in its statement.
These were found to be mild to moderate in severity and consistent with reactions that have been reported in adults.”
The study reportedly showed FIRAZYR was “well tolerated” and demonstrated rapid resolution of symptoms during an HAE attack through a single injection.
Shire said the study that led to the approval was 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 international patient organisation HAEi.
“Clinical work to bring treatment options to younger patients is vitally important and greatly appreciated by the global HAE community.”
FIRAZYR would be available for use in paediatric patients in Europe beginning in the fourth quarter, Shire confirmed.