FDA Grants Expanded Approval to Takeda’s Hyqvia to Include Treatment of Primary Immunodeficiency in Children

Rare Daily Staff

The U.S. Food and Drug Administration granted expanded approval to Takeda’s Hyqvia to treat primary immunodeficiency in children 2-16 years old.

Primary immunodeficiency (PI) is an umbrella term to describe a group of more than 400 disorders that affect the body’s immune system, increasing susceptibility to infection. Children living with PI and their families face distinct challenges as they are more likely to get frequent and serious infections that can impact their school attendance and ability to participate in social activities. While immune globulin (IG) infusions can be effective at protecting against infections associated with a PI diagnosis, some therapy options may not be optimal for all children and their families.

Hyqvia is the only subcutaneous immune globulin (ScIG) infusion that can be administered once a month—every three or four weeks—and was first approved in the United States in 2014 for the treatment of PI in adults. It is also approved in the European Union as a replacement therapy in adults, children and adolescents with PI and with secondary immunodeficiency (SID) who suffer from severe or recurrent infections, ineffective antimicrobial treatment, and either proven specific antibody failure  or serum IgG level of <4 g/L.

“Families of children living with primary immunodeficiency may feel overwhelmed by their child’s chronic medical needs. When it comes to treatment, having choices can mean a great deal to families,” said Jorey Berry, president and chief executive officer of the Immune Deficiency Foundation. “The approval of this new PI treatment for children 2 to 16 years old offers an alternative for health care providers and families who might prefer a less frequent treatment option that can be administered subcutaneously at home, after appropriate training, or in an infusion center.”

The FDA approval of Hyqvia for the treatment of PI in pediatric patients was based on evidence from a pivotal, prospective, open-label, non-controlled phase 3 clinical trial that included 44 PI patients between the ages of 2 and 16. Data were analyzed when all subjects completed 12 months of participation (one year of observation) in the trial. The data showed no clinically meaningful differences in trough Immunoglobulin G (IgG) levels across age groups.

During the 12-month trial period, Hyqvia was shown to be efficacious with respect to the occurrence of acute serious bacterial infections (aSBIs), a primary endpoint. The mean aSBI rate per year was 0.04 and was statistically significantly lower than the predefined success rate of less than one aSBI per subject per year, favoring efficacy of Hyqvia treatment in pediatric subjects with PI diseases. The efficacy of Hyqvia in this study was further demonstrated by the overall rate of infections per subject, which is consistent with results obtained in the pivotal clinical study. Results from the interim data analysis, where all subjects completed 12 months of participation in the study, indicated similar safety profiles to adults.

Photo: Jorey Berry, president and chief executive officer of the Immune Deficiency Foundation