- HYQVIA [Immune Globulin Infusion 10% (Human) with
Recombinant Human Hyaluronidase], the Only up to Once Monthly
(every 2, 3 or 4 weeks) Subcutaneous Immunoglobulin (SCIG) Infusion
to Treat CIDP, Can Be Administered by a Healthcare Professional or
Self-Administered after Appropriate Training
- Approval Based on Phase 3 ADVANCE-CIDP 1 Study Demonstrating
a Statistically Significant Difference in Relapse Rate in Favor of
HYQVIA Versus Placebo at 6 Months
- CIDP, a Rare Neuromuscular Disorder, is a Progressive,
Relapsing Peripheral Nervous System Condition Often Leading to
Debilitating Symptoms Such as Symmetric Weakness or Loss of Feeling
in the Arms and Legs
Takeda (TSE:4502/NYSE:TAK) today announced that the U.S.
Food and Drug Administration (FDA) has approved HYQVIA® [Immune
Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase]
for the treatment of chronic inflammatory demyelinating
polyneuropathy (CIDP) as maintenance therapy to prevent the relapse
of neuromuscular disability and impairment in adults. HYQVIA first
received approval in the U.S. in 2014 for the treatment of primary
immunodeficiency (PI) in adults, which has since been expanded to
include children 2-16 years old.1
HYQVIA is the only FDA-approved combination of immunoglobulin
(IG) and hyaluronidase, which makes it a facilitated subcutaneous
immunoglobulin (SCIG) infusion. For adults with CIDP, HYQVIA can be
infused up to once monthly (every two, three or four weeks) due to
the hyaluronidase component, which facilitates the dispersion and
absorption of large IG volumes in the subcutaneous space between
the skin and the muscle. Because it is delivered subcutaneously,
HYQVIA can be administered by a healthcare professional in a
medical office, infusion center or at a patient’s home. In
addition, it can be self-administered after appropriate patient or
caregiver training.1
“With the FDA approval of HYQVIA for CIDP, which builds on our
expertise in rare neuroimmunological and neuromuscular disorders,
we can now offer a personalized maintenance treatment option for
adults with this debilitating disease,” said Giles Platford,
president of Takeda’s Plasma-Derived Therapies Business Unit.
“Research and clinical experience have shown that IG therapy is
effective as maintenance treatment in adults with CIDP, and we hope
that this approval for HYQVIA is the first of several around the
world as we strive to deliver our broad and diverse IG portfolio to
more people with complex neuroimmunological diseases.”
This approval is based on results from a randomized,
double-blinded, placebo-controlled study (ADVANCE-CIDP 1) and a
single-arm, open-label, extension study (ADVANCE-CIDP 3) that
evaluated the efficacy and safety of HYQVIA as a maintenance
therapy in adults with CIDP. The efficacy evaluation included 122
adults from ADVANCE-CIDP 1 with a confirmed diagnosis of CIDP and
who had remained on a stable dosing regimen of intravenous
immunoglobulin (IVIG) therapy for at least three months prior to
screening. The analysis of the primary endpoint demonstrated a
statistically significant difference between the relapse rates in
the HYQVIA group (N=57, 14.0%) compared to the placebo group (N=65,
32.3%) (p=0.0314). The treatment difference of -18.3% (two-sided
95% CI: -32.1%, -3.1%) indicated that HYQVIA demonstrated
superiority over placebo in preventing relapse of CIDP.1
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 reactions observed in >5% of study subjects in clinical
studies of HYQVIA for CIDP were local reactions, headache, pyrexia,
nausea, fatigue, erythema, pruritus, increased lipase, abdominal
pain, back pain, and pain in extremity.1
CIDP is a rare, acquired, immune-mediated neuromuscular disorder
affecting the peripheral nervous system.2,3 It is typically
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 is often
misdiagnosed.4 The mechanism of action of IG in the treatment of
CIDP in adults has not been fully elucidated but may include
immunomodulatory effects.1 The role of IG therapy as maintenance
treatment in CIDP has been well-established and is the
guidelines-based standard of care for this complex and
heterogeneous condition.5 However, there are aspects of IVIG
treatment that can be challenging for patients such as long
treatment duration associated with high IG volumes, potential for
venous access challenges, and infusion setting limitations.5
“While it is considered the standard-of-care for maintenance
treatment of adults with CIDP, IVIG infusions may be challenging
for some patients and their caregivers,” said Lisa Butler,
executive director, GBS-CIDP Foundation International. “We’re
excited that this therapy could offer some adults with CIDP an
alternative subcutaneous option that may address some of these
challenges and help personalize treatment.”
HYQVIA is now available as a maintenance therapy for adult
patients with CIDP in the U.S. In December 2023, Takeda announced
that the European Medicines Agency’s (EMA) Committee for Medicinal
Products for Human Use (CHMP) has recommended the approval of
HYQVIA as maintenance therapy in patients with CIDP after
stabilization with IVIG. The European Commission (EC) will consider
the CHMP positive opinion when determining the potential marketing
authorization for HYQVIA for CIDP throughout the European
Union.6
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 1 and ADVANCE-CIDP 3
ADVANCE-CIDP 1 was a Phase 3, multicenter, placebo-controlled,
double-blinded study evaluating the safety, efficacy and
tolerability of HYQVIA. The primary endpoint of the ADVANCE-CIDP 1
clinical trial was the proportion of subjects who experienced a
relapse, defined as an increase of ≥1 point relative to the
pre-subcutaneous (SC) treatment baseline score in two consecutive
adjusted Inflammatory Neuropathy Cause and Treatment (INCAT)
disability scores obtained less than seven days apart. Patients
were randomized to receive either HYQVIA or placebo at the same
dose and infusion frequency as their prior IVIG treatment (every
two, three or four weeks) for six months or until withdrawal or
relapse. Those who remained relapse free were offered HYQVIA
treatment as part of ADVANCE-CIDP 3, which was an open-label
extension clinical trial to assess the long-term safety,
tolerability and immunogenicity of HYQVIA in participants with CIDP
who completed ADVANCE-CIDP 1.
Further information about the ADVANCE-CIDP 1 and ADVANCE-CIDP 3
clinical trials is available at ClinicalTrials.gov under study
identifiers NCT02549170 and NCT02955355, respectively.
HYQVIA U.S. Indication
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.
HYQVIA U.S. Important Safety Information
WARNING: THROMBOSIS
- Thrombosis may occur with immune globulin (IG) products,
including HYQVIA. 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 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.
Contraindications
- History of anaphylactic or severe systemic hypersensitivity
reactions to human IG
- IgA-deficient patients with antibodies to IgA and a history of
hypersensitivity to human IG
- Known systemic hypersensitivity to hyaluronidase including
Recombinant Human Hyaluronidase of HYQVIA
- 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.
- 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.
- 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.
- Aseptic Meningitis Syndrome: Has been reported with use
of IG, including HYQVIA and may occur more frequently in females.
The syndrome usually begins within several hours to two days
following IG treatment. 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.
- Hemolysis: HYQVIA contains 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.
- Renal Dysfunction/Failure: Acute renal
dysfunction/failure, acute tubular necrosis, proximal tubular
nephropathy, osmotic nephrosis, and death may occur with
intravenous (IV) use of IG products, especially those containing
sucrose. 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.
- Spread of Localized Infection: Do not infuse HYQVIA into
or around an infected area due to potential risk of spreading a
localized infection.
- Transfusion-Related Acute Lung Injury: Non-cardiogenic
pulmonary edema may occur with IV administered IG. 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 is made
from human plasma, it may carry a risk of transmitting infectious
agents (e.g. viruses, other pathogens). No cases of transmission of
viral diseases or variant Creutzfeldt-Jakob disease (vCJD) 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.
Adverse Reactions
The most common adverse reactions observed in >5% of patients
in the clinical trials were: Primary
Immunodeficiency (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.
Chronic Inflammatory Demyelinating
Polyneuropathy (CIDP): local reactions, headache, pyrexia,
nausea, fatigue, erythema, pruritus, increased lipase, abdominal
pain, back pain, 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, please visit:
https://www.shirecontent.com/PI/PDFs/HYQVIA_USA_ENG.pdf
For European Union Summary of Product Characteristics, please
visit:
https://www.ema.europa.eu/en/medicines/human/EPAR/hyqvia
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.
Important Notice
For the purposes of this notice, “press release” means this
document, any oral presentation, any question and answer session
and any written or oral material discussed or distributed by Takeda
Pharmaceutical Company Limited (“Takeda”) regarding this release.
This press release (including any oral briefing and any
question-and-answer in connection with it) is not intended to, and
does not constitute, represent or form part of any offer,
invitation or solicitation of any offer to purchase, otherwise
acquire, subscribe for, exchange, sell or otherwise dispose of, any
securities or the solicitation of any vote or approval in any
jurisdiction. No shares or other securities are being offered to
the public by means of this press release. No offering of
securities shall be made in the United States except pursuant to
registration under the U.S. Securities Act of 1933, as amended, or
an exemption therefrom. This press release is being given (together
with any further information which may be provided to the
recipient) on the condition that it is for use by the recipient for
information purposes only (and not for the evaluation of any
investment, acquisition, disposal or any other transaction). Any
failure to comply with these restrictions may constitute a
violation of applicable securities laws.
The companies in which Takeda directly and indirectly owns
investments are separate entities. In this press release, “Takeda”
is sometimes used for convenience where references are made to
Takeda and its subsidiaries in general. Likewise, the words “we”,
“us” and “our” are also used to refer to subsidiaries in general or
to those who work for them. These expressions are also used where
no useful purpose is served by identifying the particular company
or companies.
Forward-Looking Statements
This press release and any materials distributed in connection
with this press release may contain forward-looking statements,
beliefs or opinions regarding Takeda’s future business, future
position and results of operations, including estimates, forecasts,
targets and plans for Takeda. Without limitation, forward-looking
statements often include words such as “targets”, “plans”,
“believes”, “hopes”, “continues”, “expects”, “aims”, “intends”,
“ensures”, “will”, “may”, “should”, “would”, “could”,
“anticipates”, “estimates”, “projects” or similar expressions or
the negative thereof. These forward-looking statements are based on
assumptions about many important factors, including the following,
which could cause actual results to differ materially from those
expressed or implied by the forward-looking statements: the
economic circumstances surrounding Takeda’s global business,
including general economic conditions in Japan and the United
States; competitive pressures and developments; changes to
applicable laws and regulations, including global health care
reforms; challenges inherent in new product development, including
uncertainty of clinical success and decisions of regulatory
authorities and the timing thereof; uncertainty of commercial
success for new and existing products; manufacturing difficulties
or delays; fluctuations in interest and currency exchange rates;
claims or concerns regarding the safety or efficacy of marketed
products or product candidates; the impact of health crises, like
the novel coronavirus pandemic, on Takeda and its customers and
suppliers, including foreign governments in countries in which
Takeda operates, or on other facets of its business; the timing and
impact of post-merger integration efforts with acquired companies;
the ability to divest assets that are not core to Takeda’s
operations and the timing of any such divestment(s); and other
factors identified in Takeda’s most recent Annual Report on Form
20-F and Takeda’s other reports filed with the U.S. Securities and
Exchange Commission, available on Takeda’s website at:
https://www.takeda.com/investors/sec-filings-and-security-reports/
or at www.sec.gov. Takeda does not undertake to update any of the
forward-looking statements contained in this press release or any
other forward-looking statements it may make, except as required by
law or stock exchange rule. Past performance is not an indicator of
future results and the results or statements of Takeda in this
press release may not be indicative of, and are not an estimate,
forecast, guarantee or projection of Takeda’s future results.
Medical Information
This press release contains information about products that may
not be available in all countries, or may be available under
different trademarks, for different indications, in different
dosages, or in different strengths. Nothing contained herein should
be considered a solicitation, promotion or advertisement for any
prescription drugs including the ones under development.
_______________________ 1 HYQVIA® [Immune
Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase]
U.S. Prescribing Information. 2 GBS CIDP Foundation International.
Voice of the Patient Report. August 26, 2022. www.gbs-cidp.org.
Accessed August 2022. 3 Dalakas MC. Nat Rev Neurol.
2011;7(9):507–17. 4 Broers MC, Bunschoten C, Nieboer D, Lingsma HF,
Jacobs BC. Eur J Neurol. 2021;28(6):2065–2073. 5 Van den Bergh P,
Van Doorn PA, Hadden RD, et al. Eur J Neurol.
2021;28(11):3556–3583. 6 European Medicines Agency. HyQvia 100
mg/mL solution for infusion for subcutaneous use Summary of Product
Characteristics.
https://www.ema.europa.eu/en/documents/product-information/hyqvia-epar-product-information_en.pdf.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240115984065/en/
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
Takeda Pharmaceutical (NYSE:TAK)
Historical Stock Chart
Von Apr 2024 bis Mai 2024
Takeda Pharmaceutical (NYSE:TAK)
Historical Stock Chart
Von Mai 2023 bis Mai 2024