OSAKA, Japan & CAMBRIDGE, Mass.– Takeda (TSE:4502/NYSE:TAK) today announced that the U.S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID® [Immune Globulin Infusion (Human) 10% solution] as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP).1 It can be used as induction therapy, which includes an induction dose followed by maintenance doses.1 For treatment of CIDP, GAMMAGARD LIQUID has not been studied in immunoglobulin-naive patients nor as maintenance therapy for periods longer than six months.1
This milestone follows the recent FDA approval of HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] for maintenance therapy to prevent the relapse of neuromuscular disability and impairment in adults with CIDP.2 HYQVIA is the only combination of immunoglobulin (IG) and hyaluronidase, which makes it a facilitated subcutaneous IG infusion.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,” said Richard Ascroft, senior vice president and head of Takeda’s U.S. Plasma-Derived Therapies Business Unit. “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.”
The approval is based on results from a prospective, open-label, single-arm, multicenter clinical study (ADVANCE-CIDP 2) that evaluated the efficacy and safety of GAMMAGARD LIQUID in adults with CIDP who developed a relapse in the randomized, double-blinded, placebo-controlled study evaluating efficacy, safety and tolerability of HYQVIA (ADVANCE-CIDP 1) in adults with CIDP. Efficacy in ADVANCE-CIDP 2 was based on responder rate, where a responder was defined as a subject who demonstrated an improvement of functional disability. The responder rate was 94.4% (N=18, 95% CI: 74.2% to 99.0%). Improvement in grip strength and change in Rasch-built Overall Disability Scale (R-ODS) score were recorded across participants.1
The most common adverse reactions observed in ≥5% of clinical study patients were headache, pyrexia, anemia, leukopenia, neutropenia, illness, blood creatinine increased, dizziness, migraine, somnolence, tremor, nasal dryness, abdominal pain upper, vomiting, chills, nasopharyngitis and pain in extremity.1
CIDP is a rare, acquired, immune-mediated neuromuscular disorder affecting the peripheral nervous system.3,4 It is characterized by progressive, symmetric symptoms such as weakness, tingling or loss of feeling in distal and proximal limbs, loss of reflexes and difficulty walking.3 Because its symptoms may overlap with other rare, neuromuscular conditions, CIDP may be misdiagnosed.5 The mechanism of action of immunoglobulins in the treatment of CIDP in adults has not been fully elucidated but may include immunomodulatory effects.1
“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 is the only IVIG with multiple neuromuscular disorder indications in the U.S. since it is now approved for CIDP and it is the only FDA-approved IVIG to treat multifocal motor neuropathy as a maintenance therapy to improve muscle strength and disability in adults.1 It is also indicated in the U.S. as a replacement therapy for people two years of age or older living with primary immunodeficiency.1
References
1 GAMMAGARD LIQUID® [Immune Globulin Infusion (Human) 10% solution] U.S. Prescribing Information. https://www.shirecontent.com/PI/PDFs/GAMLIQUID_USA_ENG.pdf
2 HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] U.S. Prescribing Information. https://www.shirecontent.com/PI/PDFs/HYQVIA_USA_ENG.pdf
3 Dalakas MC. Nat Rev Neurol. 2011;7(9):507–17.
4 GBS CIDP Foundation International. Voice of the Patient Report. August 26, 2022. www.gbs-cidp.org. Accessed August 2022.
5 Broers MC, Bunschoten C, Nieboer D, Lingsma HF, Jacobs BC. Eur J Neurol. 2021;28(6):2065-2073.
