Responder Rate Data from ADVANCE-CIDP 2 Trial Leads to FDA Approval of Gammagard Liquid for CIDP

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The FDA has approved Takeda’s intravenous immunoglobulin Gammagard Liquid [immune globulin infusion (human) 10% solution] for the treatment of neuromuscular disability and impairment in adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP).¹ The regulatory action is the second approval in CIDP this month for Takeda after the FDA gave the greenlight to HyQvia [immune globulin infusion 10% (human) with recombinant human hyaluronidase] as a maintenance therapy to protect against relapse of neuromuscular disability and impairment in patients with CIDP.²

“The approval of Gammagard Liquid for treatment of CIDP is an encouraging validation of our decades-long commitment to advancing plasma-derived therapies on behalf of people living with rare neuromuscular disorders and bringing our portfolio of differentiated IG therapies to these patients,” Richard Ascroft, Takeda senior vice president and head of the US Plasma-Derived Therapies Business Unit, said in a press release. “Together with the recent Hyqvia approval in the US, we can now offer induction and maintenance therapy options to adults living with CIDP that may accommodate their personal treatment needs.”¹

HyQvia, the only subcutaneous immune globulin infusion that can be administered once monthly, was initially approved by the FDA in 2014 for the treatment of PI in adults.²

Gammagard Liquid has been approved by the FDA to treat neuromuscular disability and impairment in adults with CIDP; as a replacement treatment for primary immunodeficiency (PI) in patients aged 2 years and older; and as a maintenance treatment to improve muscle strength and disability in adults with multifocal motor neuropathy. For CIDP, use of Gammagard Liquid has not been evaluated in immunoglobulin-naive patients nor for maintenance treatment lasting longer than six months.

The FDA based its approval on data from the prospective, open-label, single-arm, multicenter ADVANCE-CIDP 2 (NCT02549170) clinical trial. The study analyzed the efficacy and safety of Gammagard Liquid in 18 adult patients with CIDP who relapsed after treatment with Hyqvia in the randomized, double-blind, placebo-controlled ADVANCE-CIDP 1 trial. Investigators administered Gammagard Liquid at an induction dose of 2 g/kg body weight, followed by maintenance infusions every three weeks for six months, with dosing adjusted at investigator's discretion.

Gammagard Liquid’s efficacy was shown based on a responder rate of 94.4% (N=18, 95% CI: 74.2% to 99.0%), with responders defined as those who achieved improved functional disability in the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score. All patients enrolled in ADVANCE-CIDP 2 showed improved functional ability defined by composite outcome metrics, which included INCAT score, grip strength, and Rasch-built Overall Disability Scale score.

In terms of safety, the most common adverse events reported in ≥5% of patients enrolled in the trial were headache, pyrexia, anemia, leukopenia, neutropenia, illness, blood creatinine increased, dizziness, migraine, somnolence, tremor, nasal dryness, abdominal pain upper, vomiting, chills, nasopharyngitis, and extremity pain.

“As the standard of care for the treatment of CIDP, IG therapy is thought to help normalize compromised immune systems through immunomodulatory mechanisms,” said Mamatha Pasnoor, MD, professor in the Department of Neurology at the University of Kansas Medical Center, said in the release.1 “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.”¹

References

1. Takeda’s GAMMAGARD LIQUID® Approved by U.S. FDA for Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Takeda. News release. January 29, 2024. Accessed January 30, 2024. https://www.takeda.com/newsroom/newsreleases/2024/Takedas-GAMMAGARD-LIQUID-Approved-for-Adults-with-CIDP-in-United-States/

2. U.S. FDA Approves Takeda’s HYQVIA® as Maintenance Therapy in Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP). Takeda. News release. January 16, 2024. Accessed January 30, 2024. https://www.takeda.com/en-us/newsroom/news-releases/2024/us-fda-approves-takedas-hyqvia-as-maintenance-therapy-in-adults-with-chronic-inflammatory-demyelinating-polyneuropathy-cidp