- If Approved, HYQVIA [Immune Globulin Infusion 10% (Human)
with Recombinant Human Hyaluronidase] Would Offer an up to
Once-Monthly Facilitated Subcutaneous At-Home or In-Office
Treatment Option
- Positive Opinion Based on Phase 3 ADVANCE-CIDP 1 Study,
Which Met its Primary Endpoint Demonstrating a Statistically
Significant Reduction in Relapse Rate 1
Takeda (TSE:4502/NYSE:TAK) today announced
the European Medicines Agency’s (EMA) Committee for Medicinal
Products for Human Use (CHMP) has recommended the approval of
HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant
Human Hyaluronidase] in patients with chronic inflammatory
demyelinating polyneuropathy (CIDP) as maintenance therapy after
stabilization with intravenous immunoglobulin therapy (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.
“Takeda is focused on bringing its differentiated immunoglobulin
therapies to patients with neuroimmunological disorders, providing
treatment options that address the needs of a broad range of
patients,” said Kristina Allikmets, senior vice president and head
of Research & Development for Takeda’s Plasma-Derived Therapies
Business Unit. “This positive CHMP opinion is a crucial step
towards bringing patients with CIDP and their caregivers an
effective therapy that, if approved, may offer maintenance
treatment personalization through up to once-monthly facilitated
subcutaneous administration at home or in office.”
CIDP is an acquired, immune-mediated condition affecting the
peripheral nervous system that is characterized by progressive,
symmetric weakness in distal and proximal limbs and impaired
sensory function in extremities.2 The role of IGs as maintenance
therapy for this rare, debilitating and slowly progressing or
relapsing disease has been well-established and is considered a
standard of care for this complex and heterogeneous condition in
guidelines from the European Academy of Neurology and Peripheral
Nerve Society due to its broad immunomodulatory and
anti-inflammatory effects.3,4 However, the high volume and
frequency of treatment required to effectively manage this disease
means that treatment can be a challenge for patients and their
health care providers.
This proposed extension of indication for HYQVIA is based on
data from the pivotal Phase 3 ADVANCE-CIDP 1 clinical trial, which
investigated HYQVIA as maintenance therapy in adult patients with
CIDP.
HYQVIA is also under regulatory review in the United States for
use as a maintenance therapy in adult patients with CIDP.
About HYQVIA®
HYQVIA® [Immune Globulin Infusion 10% (Human) with Recombinant
Human Hyaluronidase] is a liquid medicine containing Recombinant
Human Hyaluronidase and immunoglobulins (Ig) and is approved by the
European Medicines Agency (EMA) as a replacement therapy in adults,
children and adolescents with primary immunodeficiency (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. It is also approved in the United States to treat
adults and children two years of age and older with PI. HYQVIA is
infused under the skin into the fatty subcutaneous tissue. HYQVIA
contains immunoglobulins collected from human plasma.
Immunoglobulins are antibodies that maintain the body’s immune
system. The hyaluronidase part of HYQVIA helps more of the Ig get
absorbed into the body. HYQVIA is infused up to once a month (every
three or four weeks).
About the ADVANCE Clinical Program
ADVANCE-CIDP 1 was a Phase 3, multicenter, placebo-controlled,
double-blinded study to evaluate the efficacy, safety and
tolerability of HYQVIA® [Immune Globulin Infusion 10% (Human) with
Recombinant Human Hyaluronidase] as a maintenance therapy to
prevent relapse in chronic inflammatory demyelinating
polyneuropathy (CIDP). The global study included 132 adults 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 primary endpoint of the clinical trial was the proportion of
subjects who experienced a worsening of functional disability,
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. The primary efficacy analysis compared relapse rates using
a continuity-corrected χ2 test conducted at the 5% level of
statistical significance, with missing data imputed as no relapse.
Some of the secondary endpoints included time to relapse as defined
by relapse probability, effect on activities of daily living (ADL),
safety and tolerability. 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 relapse. Patients who relapsed were offered IVIG
treatment as rescue therapy for a period of up to six months. Those
who remained relapse free were offered to continue HYQVIA treatment
as part of ADVANCE-CIDP 3, 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 clinical trial is
available at ClinicalTrials.gov under study identifier
NCT02549170.
HyQvia® (Human normal immunoglobulin) 100 mg/ml solution for
infusion for subcutaneous use PRESCRIBING INFORMATION
Always refer to the Summary of Product
Characteristics (SmPC) and the local prescribing information
of your country before prescribing.
Presentation: HyQvia is a
dual vial unit consisting of one vial of 10% human normal
immunoglobulin (IG) and one vial of recombinant human hyaluronidase
(see the SmPC for details).
Indications: Replacement
therapy in adults, children and adolescents (0-18 years) in:
primary immunodeficiency syndromes with impaired antibody
production; secondary immunodeficiencies (SID) in patients who
suffer from severe or recurrent infections, ineffective
antimicrobial treatment and either proven specific antibody failure
(PSAF) or serum IgG level of 6 g/l). PSAF is a failure to mount at
least a 2- fold rise in IgG antibody titre to pneumococcal
polysaccharide and polypeptide antigen vaccines.
Dosage and administration:
For subcutaneous use only. Replacement therapy should be initiated
and monitored under the supervision of a physician experienced in
the treatment of immunodeficiency. The product should be brought to
room temperature before use. Inspect both vials for discolouration
and particulate matter before administration. Do not use heating
devices including microwaves. Do not shake or mix the components of
the two vials. Suggested infusion site(s) are the middle to upper
abdomen and thighs. The two components of the medicinal product
must be administered sequentially through the same needle beginning
with the recombinant human hyaluronidase followed by IG 10%. Please
see the SmPC for infusion rates. The full contents of the
recombinant human hyaluronidase vial should be administered
regardless of whether the full contents of the IG 10% vial is
administered. Longer needles may be used under medical supervision
to prevent infusion site leakage. Home treatment should be
initiated and monitored by a physician experienced in the guidance
of patients for home treatment.
Posology: Dose and dosage regimen
may need to be individualised for each patient dependent on the
response. Dose based on body weight may require adjustment in
underweight or overweight patients. Patients naïve to IG therapy:
The dose required to achieve a trough level of 6 g/l is
approximately 0.4-0.8 g/kg body weight/month. The dosage interval
to maintain steady state levels varies from 2-4 weeks. Trough
levels should be measured and assessed in conjunction with the
incidence of infection. To reduce the rate of infection, it may be
necessary to increase the dosage and aim for higher trough levels
(>6 g/l). At the initiation of therapy, it is recommended that
the treatment intervals for the first infusions be gradually
prolonged from a 1-week dose to up to a 3- or 4-week dose. Patients
previously treated with IG administered intravenously (IV): For
patients switching directly from IV IG, or who have had a previous
IV dose of IG that can be referenced, the medicinal product should
be administered at the same dose and at the same frequency as their
previous IV IG treatment. Patients previously treated with IG
administered subcutaneously: For patients currently being
administered IG subcutaneously, the initial dose of HyQvia is the
same as for subcutaneous treatment but may be adjusted to 3- or
4-week intervals. For patients switching directly from an IG
treatment administered subcutaneously, the first infusion of HyQvia
should be given one week after the last treatment with the previous
IG. Secondary immunodeficiencies: The recommended dose is 0.2-0.4
g/kg every three to four weeks. Trough levels should be measured
and assessed in conjunction with the incidence of infection. Dose
should be adjusted as necessary to achieve optimal protection
against infections, an increase may be necessary in patients with
persisting infection; a dose decrease can be considered when the
patient remains infection free. Children and adolescents (0-18
years): Follow adult dosage guidance.
Contraindications:
Hypersensitivity to any ingredient or human IG especially in
patients with antibodies against IgA; systemic hypersensitivity to
hyaluronidase or human recombinant hyaluronidase; HyQvia must not
be given IV or intramuscularly.
Warnings and precautions: If
HyQvia is accidentally administered into a blood vessel, patients
could develop shock. The recommended infusion rate given in the
SmPC should be adhered to. Infuse slowly and monitor closely
throughout the infusion period, particularly patients starting
therapy. Patients may require monitoring for up to 1 hour after
administration. Manage infusion related events by slowing the
infusion rate or stopping the infusion. Treatment will depend on
the nature and severity of the adverse event. Patients should be
reminded to report chronic inflammation and nodules which occur at
the infusion site or other locations. For home treatment, patients
should have the support of another responsible person in case of
adverse reactions. Record treatment with HyQvia and batch number in
patients’ notes.
Hypersensitivity: Hypersensitivity
reactions are possible in patients with anti-IgA antibodies who
should only be treated with HyQvia if alternative treatments are
not possible and under close medical supervision. In case of
hypersensitivity, shock or anaphylactic-like reactions, discontinue
the infusion immediately and treat the patient for shock. Rarely,
human normal IG can induce a fall in blood pressure with
anaphylactic reaction. In high-risk patients HyQvia should only be
administered where supportive care is available for life
threatening reactions. Patients should be informed of the early
signs of anaphylaxis/ hypersensitivity. Pre-medication may be used
as a preventative measure.
Hypersensitivity to recombinant human
hyaluronidase: Any suspicion of allergic or anaphylactic
like reactions following recombinant human hyaluronidase
administration requires immediate discontinuation of the infusion
and standard medical treatment should be administered, if
necessary. Immunogenicity of recombinant human hyaluronidase:
development of non-neutralising antibodies to the recombinant human
hyaluronidase component has been reported in patients receiving
HyQvia in clinical studies.
Thromboembolism: Thromboembolic
events including myocardial infarction, stroke, deep venous
thrombosis and pulmonary embolism have been observed with IG
treatment and cannot be excluded with use of HyQvia. Ensure
adequate hydration prior to treatment. Monitor for signs and
symptoms of thrombosis and assess blood viscosity in patients at
risk. Patients should be informed about initial symptoms and
advised to contact their physician immediately upon onset.
Haemolytic anaemia: IG products
contain antibodies to blood groups (e.g. A, B, D) which may act as
haemolysins. Monitor for signs and symptoms of haemolysis.
Acute renal failure: Severe renal
adverse reactions have been reported in patients receiving IV
IG.
Aseptic meningitis syndrome: has
been reported, symptoms usually begin within several hours to 2
days following treatment. Patients should be informed about initial
symptoms. Discontinuation of IG treatment may result in remission
within several days without sequelae.
Transmissible agents: Infectious
diseases due to the transmission of infective agents cannot be
totally excluded.
Sodium content: The recombinant
human hyaluronidase component contains 4.03 mg sodium/mL. To be
taken into consideration by patients on a controlled sodium
diet.
Traceability: The name and the
batch number of the administered product should be clearly
recorded.
Interactions: Live attenuated virus vaccines – postpone
vaccination for 3 months after treatment with HyQvia. For measles
vaccine, impairment may persist for up to 1 year, so check antibody
status. Please see the SmPC for details.
Fertility, pregnancy and
lactation: Safety during pregnancy has not been
established and immunoglobulins are excreted into the milk,
therefore use with caution in pregnant and breastfeeding
mothers.
Effects on ability to drive and use
machines: HyQvia has no or negligible influence on the
ability to drive and use machines, e.g. dizziness. Please see the
SmPC for details.
Undesirable effects:
Very common (≥1/10 patients): Feeling
hot, infusion site paraesthesia, infusion site pain (including
discomfort, tenderness, groin pain).
Common (≥1/100, < 1/10
patients): Vomiting, nausea, abdominal pain (including
abdominal upper and lower pain and tenderness), diarrhoea, infusion
site erythema, infusion site swelling (including local swelling and
oedema), infusion site pruritus (including vulvovaginal pruritus),
pyrexia, asthenic conditions (including asthenia, fatigue,
lethargy, malaise), myalgia, musculoskeletal chest pain,
headache.
Other serious undesirable effects (rare or
unknown frequency): Direct Coombs’ test positive, meningitis
aseptic. Refer to the SmPC for details on full side effect and
interactions.
Marketing Authorisation (MA)
numbers: 2.5g EU/1/13/840/001, 5g EU/1/13/840/002, 10g
EU/1/13/840/003, 20g EU/1/13/840/004, 30g EU/1/13/840/005. Name and
address of MA holder: Baxalta Innovations GmbH, Industriestrasse
67, A-1221 Vienna, Austria. HyQvia is a registered trade name.
PI approval code:
pi-02539
Date of preparation: June
2023.
Further information is available on request.
Adverse events should be reported to the authorities in your
country as required by local law. Adverse events should also be
reported to Takeda at: GPSE@takeda.com
This medicinal product is subject to
additional monitoring. This will allow quick identification of new
safety information. Healthcare professionals are asked to report
any suspected adverse reactions. See section 4.8. of the SmPc for
how to report adverse reactions.
For Full U.S. Prescribing Information, 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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Medical Information
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______________________________________ 1 Bril V, Hadden RDM,
Brannagan TH 3rd, Bar M, Chroni E, Rejdak K, Rivero A, Andersen H,
Latov N, Levine T, Pasnoor M, Sacconi S, Souayah N, Anderson-Smits
C, Duff K, Greco E, Hasan S, Li Z, Yel L, Ay H.
Hyaluronidase-facilitated subcutaneous immunoglobulin 10% as
maintenance therapy for chronic inflammatory demyelinating
polyradiculoneuropathy: The ADVANCE-CIDP 1 randomized controlled
trial. J Peripher Nerv Syst. 2023 Sep;28(3):436-449. doi:
10.1111/jns.12573. Epub 2023 Jul 6. PMID: 37314318. 2 Dalakas MC.
Nat Rev Neurol. 2011;7(9):507–17. 3 Eftimov F, et al. Cochrane
Database Syst Rev. 2013;12:CD001797. 4 Van den Bergh, P. Y. (2021).
European Academy of Neurology/Peripheral Nerve Society guideline on
diagnosis and treatment of chronic inflammatory demyelinating
polyradiculoneuropathy: Report of a joint task force—second
revision. European Journal of Neurology, 28(11), 3556–3583.
https://doi.org/10.1111/ene.14959
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Media: Lauren Padovan Lauren.padovan@takeda.com +1 (617)
431-8028
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