- 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 Phase 3 ADVANCE-CIDP Open-label Study
Data Demonstrating Safety and Efficacy as an Intravenous Therapy
for Adults with CIDP
- GAMMAGARD LIQUID [Immune Globulin Infusion (Human)
10% Solution] is the Only Intravenous IG (IVIG) Approved
for the Treatment of Multiple Neuromuscular Disorder Indications in
the U.S.
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
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.
Further information about the ADVANCE-CIDP 2 study is available
at ClinicalTrials.gov under study identifier NCT02549170.
INDICATIONS GAMMAGARD LIQUID is indicated as
replacement therapy for Primary Humoral Immunodeficiency (PI) in
adult and pediatric patients ≥2 years, as a maintenance therapy to
improve muscle strength and disability in adult patients with
Multifocal Motor Neuropathy (MMN), and as a therapy to improve
neuromuscular disability and impairment in adult patients with
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP).
LIMITATIONS OF USE (CIDP): GAMMAGARD LIQUID
has not been studied in immunoglobulin-naive patients with CIDP.
GAMMAGARD LIQUID maintenance therapy in CIDP has not been studied
for periods longer than 6 months. After responding during an
initial treatment period, not all patients require indefinite
maintenance therapy with GAMMAGARD LIQUID in order to remain free
of CIDP symptoms. Individualize the duration of any treatment
beyond 6 months based upon the patient’s response and demonstrated
need for continued therapy.
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 maintenance therapy to prevent relapse of
neuromuscular disability and 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: THROMBOSIS HYQVIA and GAMMAGARD
LIQUID
- 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 at risk of thrombosis, administer HYQVIA and
GAMMAGARD LIQUID at the minimum dose and infusion rate practicable.
Ensure adequate hydration in patients before
administration.
- Monitor for signs and symptoms of thrombosis and assess
blood viscosity in patients at risk of hyperviscosity.
WARNING: RENAL DYSFUNCTION and ACUTE RENAL FAILURE
GAMMAGARD LIQUID
- 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 or severe systemic
hypersensitivity reactions to human IG, and IgA-deficient patients
with antibodies to IgA and a history of hypersensitivity to human
IG. Anaphylaxis has been reported with intravenous (IV) use of
GAMMAGARD LIQUID.
- Additionally, HYQVIA is contraindicated in patients with
known systemic hypersensitivity to hyaluronidase including
Recombinant Human Hyaluronidase of HYQVIA, and known
systemic hypersensitivity to human albumin (in the hyaluronidase
solution).
Warnings and Precautions Hypersensitivity:
Severe hypersensitivity reactions may occur, even in patients who
have tolerated previous treatment with human IG. If a
hypersensitivity reaction occurs, discontinue infusion immediately
and institute appropriate treatment. IgA-deficient patients with
antibodies to IgA are at greater risk of developing potentially
severe hypersensitivity reactions, including anaphylaxis.
Renal Dysfunction/Failure: Acute renal
dysfunction/failure, acute tubular necrosis, proximal tubular
nephropathy, osmotic nephrosis, and death may occur with IV use of
IG products, especially those containing sucrose. Acute renal
dysfunction/failure has been reported in association with infusions
of GAMMAGARD LIQUID. Ensure patients are not volume depleted
prior to infusion. In patients at risk due to pre-existing renal
insufficiency or predisposition to acute renal failure, assess
renal function before initiation and throughout treatment, and
consider lower, more frequent dosing. If renal function
deteriorates, consider discontinuation.
Thrombosis: Has been reported to occur following
treatment with IG products, including HYQVIA and in the
absence of known risk factors. In patients at risk, administer at
the minimum dose and infusion rate practicable. Ensure adequate
hydration before administration. Monitor for signs and symptoms of
thrombosis and assess blood viscosity in patients at risk for
hyperviscosity.
Aseptic Meningitis Syndrome: Has been reported with use
of IG, including HYQVIA and may occur more frequently in
females. Conduct a thorough neurological exam on patients
exhibiting signs and symptoms, to rule out other causes of
meningitis. Discontinuing IG treatment has resulted in remission
within several days without sequelae. The syndrome usually begins
within several hours to two days following IG treatment.
Hemolysis: HYQVIA and GAMMAGARD LIQUID contain
blood group antibodies, which may cause a positive direct
antiglobulin reaction and hemolysis. Monitor patients for signs and
symptoms of hemolysis and delayed hemolytic anemia and, if present,
perform appropriate confirmatory lab testing.
Transfusion-Related Acute Lung Injury: Non-cardiogenic
pulmonary edema has been reported with IV-administered IG,
including GAMMAGARD LIQUID. Monitor patients for pulmonary
adverse reactions. If suspected, perform appropriate tests for
presence of anti-neutrophil and anti-HLA antibodies in both product
and patient serum. May be managed using oxygen therapy with
adequate ventilatory support.
Transmittable Infectious Agents: Because HYQVIA
and GAMMAGARD LIQUID are made from human plasma, they may
carry a risk of transmitting infectious agents (e.g., viruses,
other pathogens). No confirmed cases of viral transmission of
variant Creutzfeldt-Jakob disease (vCJD) have been associated with
GAMMAGARD LIQUID, and no cases have been associated with
HYQVIA.
Interference with Lab Tests: False positive serological
test results and certain assay readings, with the potential for
misleading interpretation, may occur as the result of passively
transferred antibodies.
Additional Warnings and Precautions for HYQVIA
Immunogenicity of Recombinant Human Hyaluronidase (rHuPH20):
Non-neutralizing antibodies to the Recombinant Human Hyaluronidase
component can develop. 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 critical to distinguish true
hyponatremia from a pseudohyponatremia because certain treatments
may lead to volume depletion, a further increase in serum
viscosity, and a predisposition to thromboembolic 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.
GAMMAGARD LIQUID The most serious adverse reactions
observed in clinical studies in PI was aseptic meningitis, and in
MMN were pulmonary embolism and blurred vision. The most common
adverse reactions observed in ≥5% of patients in the clinical
trials were: IV administration for
PI: Headache, fatigue, pyrexia, nausea, chills, rigors, pain
in extremity, diarrhea, migraine, dizziness, vomiting, cough,
urticaria, asthma, pharyngolaryngeal pain, rash, arthralgia,
myalgia, oedema peripheral, pruritus, and cardiac murmur.
Subcutaneous administration for PI:
Infusion site (local) event (rash, erythema, edema, hemorrhage, and
irritation), headache, fatigue, heart rate increased, pyrexia,
abdominal pain upper, nausea, vomiting, asthma, blood pressure
systolic increased, diarrhea, ear pain, aphthous stomatitis,
migraine, oropharyngeal pain, and pain in extremity.
IV administration for MMN: Headache,
chest discomfort, muscle spasms, muscular weakness, nausea,
oropharyngeal pain, and pain in extremity. IV administration for CIDP: 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.
Drug Interactions Passive transfer of antibodies
may transiently interfere with the immune responses to live
attenuated virus vaccines (e.g., measles, mumps, rubella, and
varicella).
Use In Specific Populations Pregnancy:
Limited human data are available on the use of HYQVIA during
pregnancy. The effects of antibodies to the Recombinant Human
Hyaluronidase on the human embryo or fetal development are unknown.
It is not known whether HYQVIA can cause fetal harm when
administered to a pregnant woman or if it can affect reproductive
capacity. HYQVIA should be given to a pregnant woman only if
clearly needed.
For Full U.S. Prescribing Information for GAMMAGARD LIQUID,
please visit:
https://www.shirecontent.com/PI/PDFs/GAMLIQUID_USA_ENG.pdf
For Full U.S. Prescribing Information for HYQVIA, please
visit:
https://www.shirecontent.com/PI/PDFs/HYQVIA_USA_ENG.pdf
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. For
more information, visit www.takeda.com.
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information about products that may not be available in all
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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 Media Lauren Padovan
Lauren.padovan@takeda.com +1 (617) 431-8028
U.S. Media Courtney Winger Courtney.winger@takeda.com +1
(617) 301-0687
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