“Why can’t Icatibant be used in children? What can happen?”

Sep 1, 2015

Dr. C: We would like to thank our reader for introducing this important topic in the management of HAE—children. The limited available pediatric treatment options comprise a serious unmet need. The FDA approved on-demand drugs include: Ecallantide, for ages 12 and older; Icatibant, for ages 18 and older; plasma derived C1 inhibitor and recombinant human C1 inhibitor for adolescents and adults. The reason that we do not have approval for the use of these drugs in children is not an issue of safety or efficacy concerns in younger age groups but rather a reflection of the difficulties drug companies face conducting trials in ‘protected populations’ such as children and pregnant women. To get their drugs approved and available for use pharmaceutical companies appropriately initially target the ‘low hanging fruit’ ie older age groups. We are grateful to have the options for treatment that are now available. We cannot rest however until these options are approved and thereby easier to obtain for all our patients—including children. Bruce what are your thoughts regarding treatment options for children with HAE?

Dr. Z: As our readers know HAE is a disease that presents in childhood. Sandra and I have been working on a manuscript that highlights the pattern that earlier onset of swelling tends to accompany greater disease severity and longer delays to correct diagnosis.  Thus, there is a compelling need to make the diagnosis of HAE at a younger age and have on-demand medicines available to treat children. While not currently approved for use in children, physicians can use medicines “off label”. I have recommended some of these medicines for children. The biggest hurdle, however, is getting them approved by the insurance companies. The limited data that is available for ecallantide and C1 inhibitor suggests that their safety and efficacy is similar to that seen in adults. There is no published data that I’m aware of regarding the use of icatibant in children, however I know that Shire is currently conducting a trial in children. Marc, any thoughts here?

Dr. R:. Icatibant is not FDA-approved for use in people under the age of 18.  This is due to a lack of safety and efficacy data of the medication in this younger population as children and adolescents were not included in the initial pivotal studies on icatibant for HAE.  So currently we just don’t have enough information to know how well it works or what side effects might occur in children.  Safety, is of course, of utmost importance in kids so we need to carefully examine this in controlled, monitored studies.  Currently, specific pediatric studies on HAE medications – including icatibant, ecallantide, C1INH and recombinant C1INH– are ongoing.  Hopefully, once completed, these will give us additional data on which HAE drugs can be used safely and effectively in younger age groups.

Dr. C:  Thank you, Bruce and Marc. I hope that this has been helpful for our readers, there is much more to be done—the health and well being of our children is our greatest priority. We look forward to our next ‘Question of the Week’.

include(TEMPLATEPATH . '/single-blog.php'); ?>