FDA Approves Expanded Indication for Hyqvia to Treat Pediatric Primary Immunodeficiency


During the 12-month trial period, Hyqvia was shown to be efficacious treating the occurrence of acute serious bacterial infections in children aged 2 through 16 years with primary immunodeficiency.

The FDA has approved a supplemental biologics license application to expand the use of immune globulin infusion 10% with recombinant human hyaluronidase (Hyqvia; Takeda) to treat primary immunodeficiency in children aged 2 through 16 years.

U.S. Food and Drug Administration FDA approved vector stamp | Image credit: Calin - stock.adobe.com

U.S. Food and Drug Administration FDA approved vector stamp | Image credit: Calin - stock.adobe.com

Hyqvia is the only subcutaneous immune globulin infusion that can be administered once per month. It was first approved in the United States in 2014 for the treatment of primary immunodeficiency in adults. Primary immunodeficiency encompasses a group of more than 400 disorders affecting the body’s immune system, increasing susceptibility to infection.

Children with primary immunodeficiency and their families have unique challenges, with frequent and serious infections impacting school attendance and the ability to participate in social activities. Although immune globulin infusions can help protect against infections associated with primary immunodeficiency, some therapy options are not optimal for all children and their families.

For example, intravenous route of administration may be difficult for some children and the frequency of some subcutaneous therapies may be difficult for some families’ schedules.

“Families of children living with primary immunodeficiency may feel overwhelmed by their child’s chronic medical needs,” said Jorey Berry, president and chief executive officer of the Immune Deficiency Foundation, in a press release. “When it comes to treatment, having choices can mean a great deal to families.”

The new approval is based on evidence from a pivotal, prospective, open-label, non-controlled phase 3 clinical trial including 44 patients with primary immunodeficiency between the ages of 2 and 16 years. Data were analyzed when all subjects completed 12 months of participation in the trial.

According to the data, there were 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 1 aSBI per subject per year, favoring the efficacy of Hyqvia in pediatric subjects with primary immunodeficiency. The efficacy of Hyqvia was further demonstrated by the overall rate of infections per subject, which was consistent with results obtained in the pivotal clinical study. The mean rate of all infections per subject-year was 3.20, with an upper limit of the 95% confidence interval of 4.05.

Results from the interim data analysis, in which all subjects completed 12 months of participation in the study, indicated similar safety profiles to adults.

“The approval of this new [primary immunodeficiency] 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,” Berry said in the press release.

Hyqvia is a liquid medication that is infused under the skin into the fatty subcutaneous tissue. It contained immunoglobulins collected from human plasma, which maintain the body’s immune system. The hyaluronidase in Hyqvia helps more of the immunoglobulin absorb into the body.

“This expanded Hyqvia indication exemplifies our ongoing commitment to providing plasma-derived therapies with proven efficacy,” said Brandon Monk, MBA, head of Takeda’s United States Plasma-Derived Therapies Business Unit, in the press release. “Hyqvia is now available to a broader community impacted by [primary immunodeficiency], including children and their families with distinct needs, who may prefer flexible treatment options in the management of these disorders.”


Takeda Receives FDA Approval to Expand the Use of Hyqvia to Treat Primary Immunodeficiency in Children. News release. Takeda. April 11, 2023. Accessed April 12, 2023. https://www.takeda.com/en-us/newsroom/news-releases/2023/takeda-receives-fda-approval-to-expand-the-use-of-hyqvia

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