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New Indication Delivers on Takeda’s Commitment to Expanding its Broad and Diverse Immunoglobulin (IG) Portfolio to Meet the Needs of People Living with CIDP
Approval Supported by Data from the Phase 3 Open-Label ADVANCE-CIDP Study Demonstrating Safety and Efficacy as Intravenous Therapy for Adults With PRIC
GAMMAGARD LIQUID [10% Human Immunoglobulin Infusion Solution] is the only intravenous GI (IVIG) approved for the treatment of multiple indications of neuromuscular disorders in the United States.
OSAKA, Japan and CAMBRIDGE, Mass. , Jan. 29, 2024–(BUSINESS WIRE)–Takeda (TSE: 4502/NYSE: TAK) today announced that the U. S. Food and Drug Administration (FDA) has approved GAMMAGARD LIQUID® [10% Immune Globulin Infusion Solution (Human)] as intravenous immunoglobulin (IVIG) therapy to improve disability and neuromuscular impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). 1 It can be used as a cure for induction, which includes an induction dose. followed for a maintenance dose. 1 For the treatment of ICDP, GAMMAGARD LIQUID has not been studied in immunoglobulin-naïve patients or as a maintenance cure for periods longer than six months. 1
This milestone follows the FDA’s recent approval of HYQVIA [10% immunoglobulin (human) infusion with recombinant human hyaluronidase] as a maintenance treatment to prevent relapses of disability and neuromuscular impairment in adults with IPD. 2 HYQVIA® is the only mixture of immunoglobulin (IG) and hyaluronidase, making it a simple 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 the effects of a prospective, open-label, single-arm, multicenter clinical study (ADVANCE-CIDP 2) that evaluated the efficacy and protection of GAMMAGARD LIQUID in relapsed adults with CIDP in a randomized, two-fold study. A blinded, placebo-controlled study comparing the efficacy, protection, and tolerability of HYQVIA (ADVANCE-CIDP 1) in adults with CIDP. Efficacy in ADVANCE-CIDP 2 was based on responder rate, with one responder explained as a topic demonstrating an improvement in its functionality. disability. The response rate was 94. 4% (N=18, 95% CI 74. 2% to 99. 0%). Improvements in grip and replacement strength were recorded in participants on the constructed Rasch R-ODS (General Disability Scale) score. 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 indications for neuromuscular disorders in the United States, as it is now approved for PDIC and is the only IVIG approved by the FDA to treat multifocal motor neuropathy as a maintenance cure to improve muscle strength and disability in adults. 1 It is also indicated in the United States as the treatment of choice for others two years of age and older living with immunodeficiency number one. 1
About GAMMAGARD LIQUID GAMMAGARD LIQUID® is an intravenous immunoglobulin (IVIG) that is infused into the veins. GAMMAGARD LIQUID is approved in the United States as a GI remedy to improve disability and neuromuscular impairment in adult patients with CIDP, as the remedy of choice for number one immunodeficiency (PI) in adult and pediatric patients two years of age and older, and as maintenance therapy. A study is aimed at improving muscle strength and disability in adult patients with multifocal motor neuropathy (MMN). Also known as KIOVIG in the United States and Canada, it is approved in 66 countries around the world.
About HYQVIA HYQVIA® [Infusion of 10% Immunoglobulin (Human) with Recombinant Human Hyaluronidase] is a liquid medication containing recombinant human hyaluronidase and immunoglobulins (IGs) and is approved in the United States to treat adults and children two years of age and older with immunodeficiency (PI) and as a maintenance cure to prevent relapses of disability and neuromuscular impairment in adult patients with IDP. It is also approved through the European Medicines Agency (EMA) as a remedy of choice in adults, young people and adolescents with PI and secondary immunodeficiency (SID) who suffer from severe or recurrent infections, useless antimicrobial treatment and show express antibody failure (PSAF) or serum IgG point <4 g/L. HYQVIA is infused under the skin into subcutaneous adipose tissue. HYQVIA comprises GIs extracted from human plasma. GIs are antibodies that support the body's immune system. The hyaluronidase component of HYQVIA facilitates the dispersion and absorption of GI in the subcutaneous area between the skin and muscle. HYQVIA is infused up to once a month (every two, three or four weeks for PDIC; every three to four weeks for PI).
About ADVANCE-CIDP 2 ADVANCE-CIDP 2 was a multicenter, prospective, open-label, single-arm clinical study that evaluated the efficacy and protection of GAMMAGARD LIQUID in adults with RACCID who relapsed in the randomized, double-blind placebo group. -Controlled study comparing the efficacy, protection and tolerability of HYQVIA (ADVANCE-CIDP 1) in adults with PRI. All 18 patients who relapsed in the ADVANCE-CIDP 1 study were enrolled in the ADVANCE-CIDP 2 study.
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.
More information about ADVANCE-CIDP 2 can be found in ClinicalTrials. gov with ID NCT02549170.
INDICATIONS GAMMAGARD LIQUID is indicated as a replacement cure for humoral immunodeficiency (PI) number one in adult and pediatric patients ≥ 2 years of age, as a maintenance cure to improve muscle strength and disability in adult patients with multifocal motor neuropathy (MND), and as a remedy to improve disability and neuromuscular impairment in adult patients with chronic inflammatory demyelinating polyneuropathy (PRI).
LIMITATIONS OF USE (PRI): GAMMAGARD LIQUID has not been studied in patients with IPD who have not previously received immunoglobulins. The maintenance cure of GAMMAGARD LIQUID in PDIC has not been studied for periods longer than 6 months. After responding to an initial treatment period, not all patients require indefinite treatment. Maintenance treatment with GAMMAGARD LIQUID to avoid the symptoms of PDIC. Individualize the duration of any treatment beyond 6 months based on the patient’s reaction and demonstrated need to continue treatment.
HYQVIA is indicated for the treatment of primary immunodeficiency (PI) in adults and pediatric patients two years of age and older and for chronic inflammatory demyelinating polyneuropathy (CIDP) as a maintenance cure to prevent relapses of disability and neuromuscular impairment in adults.
HYQVIA is for subcutaneous use only. GAMMAGARD LIQUID for PI is for intravenous or subcutaneous use. GAMMAGARD LIQUID for MMN and CIDP is for intravenous use only.
IMPORTANT SAFETY INFORMATION
WARNING: THROMBOSHYQVIA and GAMMAGARD FLUID
Thrombosis may occur with immune globulin (IG) products, including HYQVIA and GAMMAGARD LIQUID. Risk factors may include advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors. Thrombosis may occur in the absence of known risk factors.
For patients threatened with thrombosis, administer HYQVIA and GAMMAGARD LIQUID at the minimum conceivable dose and infusion rate. Ensure that patients are sufficiently hydrated prior to administration.
Monitor thrombosis symptoms and symptoms and assess blood viscosity in patients with hyperviscosity.
WARNING: RENAL DYSFUNCTION and ACUTE RENAL FAILURE GAMMAGARD FLUID
Renal dysfunction, acute renal failure, osmotic nephrosis, and death may occur in predisposed patients with immune globulin intravenous (IGIV) products, including GAMMAGARD LIQUID. Patients predisposed to renal dysfunction include those with any degree of pre-existing renal insufficiency, diabetes mellitus, age greater than 65, volume depletion, sepsis, paraproteinemia, or patients receiving known nephrotoxic drugs. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. GAMMAGARD LIQUID does not contain sucrose.
Contraindications
HYQVIA and GAMMAGARD LIQUID are contraindicated in patients with a history of anaphylactic reactions or severe systemic hypersensitivity reaction to human GIs, such as in IgA deficient patients with anti-IgA antibodies and a history of hypersensitivity reaction to human GIs. On-womb anaphylaxis has been reported with intravenous (IV) use of GAMMAGARD LIQUID.
In addition, HYQVIA is contraindicated in patients with known systemic hypersensitivity reaction to hyaluronidase, recombinant human hyaluronidase of HYQVIA, and known systemic hypersensitivity reaction to human albumin (in hyaluronidase solution).
Warnings and Precautions Hypersensitivity: Severe hypersensitivity reactions are possible, even in patients who have tolerated prior human IG therapy. In case of a hypersensitivity reaction, discontinue the infusion and initiate appropriate therapy. Patients with IgA deficiency and who have anti-IgA antibodies are at increased risk of causing potentially serious hypersensitivity reactions, in addition to anaphylaxis.
Renal dysfunction/failure: Acute renal failure/failure, acute tubular necrosis, proximal tubular nephropathy, osmotic nephrosis, and death may occur with intravenous use of gastrointestinal products, especially those containing sucrose. Acute renal failure/dysfunction has been reported in conjunction with infusions of GAMMAGARD LIQUID. Make sure the patient’s volume is not exhausted prior to the infusion. In patients at risk due to pre-existing renal failure or predisposition to acute renal failure, evaluate renal service as prior to initiation and all treatment. and a lower, more common dose. If kidney function deteriorates, treatment should be stopped.
Thrombosis: Cases have been reported following treatment with gastrointestinal products, with the addition of HYQVIA and in the absence of known threat factors. In at-risk patients, administer at the lowest conceivable dose and infusion rate. Make sure there is sufficient hydration before administration. Monitor thrombosis symptoms and symptoms and assess blood viscosity in patients with hyperviscosity threat.
Aseptic meningitis syndrome: has been reported with the use of GI, adding HYQVIA, and would possibly occur more in women. Perform a complete neurological examination in patients with symptoms and symptoms, to rule out other reasons for meningitis. Discontinuation of IG treatment resulted in remission within a few days with no sequelae. The syndrome usually begins within a few hours to two days after IG treatment.
Hemolysis: HYQVIA and GAMMAGARD LIQUID involve antibodies bound to blood group, which can cause a positive direct antiglobulin reaction and hemolysis. Monitor patients for symptoms and symptoms of hemolysis and late hemolytic anemia and, if necessary, perform appropriate confirmatory laboratory tests.
Transfusion-Related Acute Lung Injury: Noncardiogenic pulmonary edema has been reported with GIs administered intravenously, with the addition of GAMMAGARD LIQUID. Monitor patients for pulmonary adverse reactions. If suspected, perform appropriate testing for the presence of antineutrophils and antineutrophils. -HLA antibodies in the serum of the product and the patient. They can be controlled with oxygen treatment with sufficiently good ventilatory support.
Transmissible infectious agents: Because HYQVIA and GAMMAGARD LIQUID are manufactured from human plasma, they may pose a threat of transmission of infectious agents (e. g. e. g. , viruses and other pathogens). There have been no reported cases of viral transmission of variant Creutzfeldt-Jakob disease (vCJD). have been linked to GAMMAGARD LIQUID and no cases have been linked to HYQVIA.
Interference with laboratory tests: False-positive effects on serological tests and safe tests are possible, with the potential for misleading interpretations, due to passively transferred antibodies.
Additional Warnings and Precautions Regarding HYQVIA Immunogenicity of Recombinant Human Hyaluronidase (rHuPH20): Non-neutralizing antibodies may develop against the recombinant human hyaluronidase component. The clinical significance of these antibodies or whether they interfere with fertilization in humans is unknown.
Spread of Localized Infection: Do not infuse HYQVIA into or around an infected area due to potential risk of spreading a localized infection.
Additional Warnings and Precautions for GAMMAGARD LIQUID Hyperproteinemia, increased serum viscosity, and hyponatremia may occur. It is to distinguish true hyponatremia from pseudohyponatremia, as some remedies could lead to a decrease in volume, a further increase in serum viscosity, and a predisposition to thromboembolic diseases. Events.
Adverse Reactions HYQVIA The most common adverse reactions observed in >5% of patients in the clinical trials were: PI: local adverse reactions including pain, erythema, edema, and pruritus, and systemic adverse reactions including, headache, antibody formation against Recombinant Human Hyaluronidase (rHuPH20), fatigue, nausea, pyrexia, and vomiting. CIDP: local reactions, headache, pyrexia, nausea, fatigue, erythema, pruritus, increased lipase, abdominal pain, back pain, and pain in extremity.
The maximum serious adverse reactions observed in the clinical studies of PI were aseptic meningitis and, in the MMN study, pulmonary embolism and blurred vision. The maximum non-unusual adverse reactions observed in ≥ 5% of patients in clinical trials were: Intravenous management for PI: Headache, fatigue, fever, nausea, chills, chills, pain in the extremities, diarrhea, migraine, dizziness, vomiting, cough, hives, asthma, pharyngolaryngeal pain, rash, arthralgia, myalgia, peripheral edema, pruritus, and central murmur. Subcutaneous management for PI: occasion of infusion (local) (rash, erythema, edema, hemorrhage and irritation), headache, fatigue, higher center rate, pyrexia, upper abdominal pain, nausea, vomiting, asthma, higher systolic blood pressure, diarrhea. , ear pain, aphthous stomatitis, migraine, oropharyngeal pain, and limb pain. Intravenous management for MMN: headache, chest discomfort, muscle spasms, muscle weakness, nausea, oropharyngeal pain, and pain in the extremities. Intravenous administration for ICDP: headache, pyrexia, anemia, leukopenia, neutropenia, disease, elevated blood creatinine levels, dizziness, migraine, drowsiness, tremors, dry nose, upper abdominal pain, vomiting, chills, nasopharyngitis, and pain in the extremities.
Passive movement of antibodies would possibly transiently interfere with immune responses to live attenuated vaccines (e. g. e. g. , measles, mumps, rubella, and chickenpox).
Use in express populations Pregnancy: There is limited human knowledge about the use of HYQVIA during pregnancy. The effects of antibodies to recombinant human hyaluronidase on the human embryo or fetal progression are unknown. It is not known whether HYQVIA can harm the fetus when given to a pregnant woman or if it would possibly affect her reproductive ability. HYQVIA deserves to be administered to a pregnant woman only when it is surely necessary.
For recipes for GAMMAGARD LIQUID across the U. S. , visit: https://www. shirecontent. com/PI/PDFs/GAMLIQUID_USA_ENG. pdf
For HYQVIA recipes across the U. S. , visit: https://www. shirecontent. com/PI/PDFs/HYQVIA_USA_ENG. pdf
About Takeda Takeda aims to create better health for people and a better future for the world. Our goal is to identify and deliver life-changing treatments in our core areas of healing and promotion, including gastrointestinal and inflammatory diseases, 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 functions through our dynamic and varied range. As a leading values-based research and development company headquartered in Japan – a biopharmaceutical company driven by
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_________________________ 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.
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International MediaLauren PadovanLauren. padovan@takeda. com 1 (617) 431-8028
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