The Food and Drug Administration (FDA) has expanded the approval of HyQvia® (immune globulin [IG] infusion 10% [human] with recombinant human hyaluronidase) to include children 2 to 16 years of age with primary immunodeficiency (PI). Previously, the treatment was only approved for adults.
HyQvia is a subcutaneous immune globulin treatment that can be administered once a month (every 3 or 4 weeks). The IG component is prepared from large pools of human plasma consisting of at least 98% IgG, and contains a broad spectrum of antibodies that provides the therapeutic effect. The recombinant human hyaluronidase component increases dispersion and absorption of the IG component.
The approval in pediatric patients was based on data from a prospective, open-label, non-controlled phase 3 trial (ClinicalTrials.gov Identifier: NCT03277313) that evaluated the efficacy and safety of HyQvia in 44 patients 2 to 16 years of age with PI who had received intravenous immunoglobulin or subcutaneous immunoglobulin treatment prior to enrollment. Data were analyzed when all patients completed 12 months of participation in the trial. The median number of infusions per month was 1.1 (range, 1.0 to 1.5) and was comparable across the age groups.
The primary analysis for efficacy was based on the rate of acute serious bacterial infections (aSBIs), defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess, per patient per year.
Results showed that the mean aSBI rate per year was 0.04 (upper 1-sided 99% CI of 0.21; P <.001), which met the predefined success rate of less than 1 aSBI per patient per year. The mean rate of all infections per patient-year was 3.20, with an upper limit of the 95% CI of 4.05. The overall rate of infections per patient was consistent with results obtained in the pivotal clinical study. Pediatric patients missed a mean of 5 days of work/school/daycare.
HyQvia is available in a dual vial unit of 2 single-use vials containing the active IG 10% and recombinant human hyaluronidase: 2.5g/200U, 5g/400U, 10g/800U, 20g/1600U, and 30g/2400U.
- Takeda receives FDA approval to expand the use of HyQvia® to treat primary immunodeficiency in children. News release. Takeda. Accessed April 11, 2023. https://www.businesswire.com/news/home/20230410005423/en/Takeda-Receives-FDA-Approval-to-Expand-the-Use-of-HYQVIA%C2%AE-to-Treat-Primary-Immunodeficiency-in-Children.
- HyQvia. Package insert. Takeda; 2023. Accessed April 11, 2023. https://www.shirecontent.com/PI/PDFs/HYQVIA_USA_ENG.pdf.