Gammagard Liquid Receives FDA Approval to Treat Disability and Impairment Associated with Chronic Inflammatory Demyelinating Polyneuropathy
Gammagard Liquid (Immune Globulin Infusion [human] 10% solution; Takeda, Cambridge, MA) has been approved by the Food and Drug Administration (FDA) as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). Gammagard Liquid is provided as an initial induction dose over 2 to 5 consecutive days followed by maintenance doses every 3 weeks. This medication previously was approved as a treatment for primary humoral immunodeficiency in adult and pediatric patients >2 years and as maintenance therapy to improve strength and disability in adults diagnosed with multifocal motor neuropathy.
The FDA approval of Gammagard Liquid for CIDP is based on data from the phase 3 ADVANCE-CIDP 2 clinical trial (NCT02549170). ADVANCE-CIDP 2 was a prospective, open-label, single-arm, multicenter study investigating the safety, tolerability, and efficacy of Gammagard Liquid as a therapy for adults with CIDP. Enrollment was offered to the 18 participants who previously relapsed during ADVANCE-CIDP 1 (NCT02549170), a phase 3 clinical trial evaluating the use of HyQvia (Immune Globulin Infusion 10% [Human] with Recombinant Human Hyaluronidase; Takeda, Cambridge, MA) as a maintenance treatment for CIDP. Participants in ADVANCE-CIDP 2 received Gammagard Liquid at an induction dose of 2g/kg body weight followed by maintenance infusions every 3 weeks for 6 months with dosing adjusted at investigators’ discretion. The primary outcome measure for the efficacy of Gammagard Liquid was responder rate, defined as demonstrated improvement in participant’s functional disability.
According to a statement from Takeda, the responder rate for the participant population (N=18) was 94.4% (95% CI, 74.2% to 99.0%). Additionally, participants showed improvements in assessed secondary outcome measures, including hand grip strength and change in Rasch-built Overall Disability Scale (R-ODS) score. The most common adverse reactions observed in ≥5% of participants were headache, pyrexia, neutropenia, illness, increase in blood creatinine, dizziness, migraine, somnolence, tremor, nasal dryness, upper abdominal pain, vomiting, chills, nasopharyngitis, and pain in extremity.
“As the standard of care for the treatment of CIDP, IG therapy is thought to help normalize compromised immune systems through immunomodulatory mechanisms,” said Dr. Mamatha Pasnoor, Professor in the Department of Neurology at the University of Kansas Medical Center. “Because CIDP is a progressive and complex disease, multiple treatment options are needed, and clinicians now have an additional therapy that can help adults with CIDP manage their disease.”
Gammagard Liquid has not been evaluated for people who are immunoglobulin-naïve nor for maintenance therapy periods longer than 6 months. The label for Gammagard Liquid contains a boxed warning for thrombosis, renal dysfunction, and acute renal failure.
Dr. Mamatha Pasnoor