“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 patients living with rare neuromuscular disorders and bringing our portfolio of differentiated IG therapies to these patients,” Richard Ascroft, JD, senior vice president and head of Takeda’s U.S. Plasma-Derived Therapies Business Unit, said in a statement.1 “Together with the recent Hyqvia approval in the U.S., we can now offer induction and maintenance therapy options to adults living with CIDP that may accommodate their personal treatment needs.”
Earlier this month, the FDA approved the company's Hyqvia, which is immune globulin (IG) infusion 10% (human) with recombinant human hyaluronidase, as maintenance therapy for adults CIDP to prevent relapse of neuromuscular disability and impairment. The approval was based on findings from ADVANCE-CIDP 1 and the single-arm, open-label, extension ADVANCE-CIDP 3 study (NCT02955355) that assessed the efficacy and safety of Hyqvia in adults with CIDP. The analysis of the primary end point among 122 with CIDP from ADVANCE-CIDP 1 showed a statistical difference between the relapse rates in the Hyqvia group (n = 57; 14.0%) compared with the placebo group (n = 65; 32.3%), which was significant (P = .0314).2
The efficacy assessment on Hyqvia included adults with a confirmed diagnosis of CIDP and who had remained on a stable dosing regimen of intravenous immunoglobulin (IVIG) therapy for at least 3 months before screening. The treatment difference of -18.3% (2-sided 95% CI, -32.1% to -3.1%) indicated that Hyqvia displayed superiority over placebo in preventing relapse of CIDP. The safety of Hyqvia in adults with CIDP was evaluated across ADVANCE-CIDP 1 (n = 62) and ADVANCE-CIDP 3 (n = 79). The most common adverse effects (AEs) observed in at least 5% of study participants in the clinical trials of Hyqvia for CIDP included local reactions, headache, pyrexia, nausea, fatigue, erythema, pruritus, increased lipase, abdominal pain, back pain, and pain in extremity.
Presented at the 2023 Peripheral Nerve Society (PNS) Annual Meeting in Copenhagen, Denmark, on June 20, 2023, data from ADVANCE-CIDP 1 also showed a lower probability of functional worsening rates with Hyqvia in comparison with placebo (37.5% vs 54.4%; 95% CI, –33.02 to 0.69).3 Patients on active therapy experienced longer time to relapse compared with those on placebo, with Kaplan-Meier estimate curves separating early, at approximately week 4. Changes on Rasch-built Overall Disability Scale centile score, a secondary end point, also continued to favor the Hyqvia group (least squares mean difference, –6.1 [SD, 1.64] vs –0.9 [SD, 1.69], respectively).
From the analysis presented at the meeting, the safety profile of Hyqvia was generally consistent with the existing EU Summary of Product Characteristics, with the most common casually-related local AEs being injection and infusion site pain and erythema, and infusion site edema and pruritis. The most common causally-related systemic AEs included headache, nausea, fatigue, and pruritus. Throughout the trial, the majority (88.7%) of patients receiving active drug were on a 4-week dosing interval with a mean time to deliver treatment of 125.9 minutes. The majority (86.3%) of patients received study treatment using 2 infusion sites per treatment, while 9.6% and 3.7% used 1 and 3 infusion sites, respectively.
The approval marks a major step forward for the CIDP treatment paradigm, which has long sought a novel therapy. In a recent interview with NeurologyLive®, Richard Lewis, MD, director of the electromyography laboratory and professor of neurology at Cedars-Sinai Medical Center, discussed how the approval marks a significant advancement in the field. He also talked about the advantages Hyqvia offers over traditional intravenous immunoglobulin treatments for patients with CIDP. Lewis, also a consultant for Takeda, spoke about how the potential shift to a 20% solution might impact the future of CIDP therapy and patient convenience.
