Takeda’s GAMMAGARD LIQUID® Approved by U.S. FDA for Adults with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

OSAKA, Japan & CAMBRIDGE, Mass. — Takeda 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). It can be used as induction therapy, which includes an induction dose followed by maintenance doses. For treatment of CIDP, GAMMAGARD LIQUID has not been studied in immunoglobulin-naive patients nor as maintenance therapy for periods longer than six months.

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. HYQVIA is the only combination of immunoglobulin (IG) and hyaluronidase, which makes it a facilitated subcutaneous IG infusion.

“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. 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. Because its symptoms may overlap with other rare, neuromuscular conditions, CIDP may be misdiagnosed. The mechanism of action of immunoglobulins in the treatment of CIDP in adults has not been fully elucidated but may include immunomodulatory effects.

“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.

 

About GAMMAGARD LIQUID 
GAMMAGARD LIQUID^® [Immune Globulin Infusion (Human) 10% solution] is an intravenous immunoglobulin (IVIG) that is infused into the veins. GAMMAGARD LIQUID is approved in the U.S. as an IG therapy to improve neuromuscular disability and impairment in adult patients with CIDP, as a replacement therapy for primary immunodeficiency (PI) in adult and pediatric patients two years of age and older, and as a maintenance therapy to improve muscle strength and disability in adult patients with multifocal motor neuropathy (MMN). Also known as KIOVIG outside the U.S. and Canada, it is approved in 66 countries worldwide.

 

About HYQVIA 
HYQVIA^® [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] is a liquid medicine containing Recombinant Human Hyaluronidase and immunoglobulin (IG) and is approved in the U.S. to treat adults and children two years of age and older with primary immunodeficiency (PI), and as maintenance therapy to prevent relapse of neuromuscular disability and impairment in adult patients with CIDP. It is also approved by the European Medicines Agency (EMA) as a replacement therapy in adults, children and adolescents with PI and with secondary immunodeficiency (SID) who suffer from severe or recurrent infections, ineffective antimicrobial treatment, and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/L. HYQVIA is infused under the skin into the fatty subcutaneous tissue. HYQVIA contains IG collected from human plasma. IG are antibodies that maintain the body’s immune system. The hyaluronidase part of HYQVIA facilitates the dispersion and absorption of IG in the subcutaneous space between the skin and the muscle. HYQVIA is infused up to once a month (every two, three or four weeks for CIDP; every three or four weeks for PI).

 

About ADVANCE-CIDP 2 
ADVANCE-CIDP 2 was a prospective, open-label, single-arm, multicenter clinical study 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. The 18 patients who relapsed during ADVANCE-CIDP 1 were offered enrollment in ADVANCE-CIDP 2.

GAMMAGARD LIQUID was administered at an induction dose of 2 g/kg body weight, followed by maintenance infusions at every three weeks for a period of six months. The dose of GAMMAGARD LIQUID treatment could be adjusted at the investigator’s discretion. Adjustments to the dosing interval of every three weeks were not allowed. All subjects completed the study. All dosed subjects were analyzed for efficacy and safety. Efficacy of ADVANCE-CIDP 2 was based on responder rate, where a responder is defined as subjects who had at least a one-point decrease in the adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) disability score at the completion of the treatment period (six months). The responder rate was 94.4% (N=18, 95% CI: 74.2% to 99.0%). The INCAT score returned to baseline values prior to joining the study in 17 of the 18 subjects (94.4%) at six months. All subjects had improvement in functional ability as defined by a composite outcome metrics that included INCAT score, grip strength, or Rasch-built Overall Disability Scale (R-ODS) score.

 

About Takeda 
Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries.