Takeda Presents Long-Term Data from Phase 3 ADVANCE-CIDP 3 Clinical Trial of HYQVIA® in Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) at PNS Annual Meeting
Takeda has shared long-term data from the Phase 3 ADVANCE-CIDP 3 clinical trial, evaluating HYQVIA for treating chronic inflammatory demyelinating polyneuropathy (CIDP). The study, the longest of its kind, demonstrated HYQVIA's safety, tolerability, and low relapse rate, supporting its use as maintenance therapy. Presented at the PNS Annual Meeting, HYQVIA is the first facilitated subcutaneous immunoglobulin for CIDP, approved by the FDA and European Commission. The trial involved 85 patients over 33 months, reporting consistent safety profiles with no new concerns. Adverse events were mostly mild or moderate, with serious events in three patients. Results indicated stable disease maintenance with a low annualized relapse rate of 4.5%.
- HYQVIA demonstrated favorable long-term safety and tolerability.
- Low relapse rate of 4.5% supports HYQVIA's efficacy as a maintenance therapy for CIDP.
- HYQVIA is the first and only facilitated subcutaneous immunoglobulin approved for CIDP.
- FDA and European Commission have approved HYQVIA for maintenance therapy in CIDP.
- HYQVIA can be self-administered or administered by healthcare professionals.
- HYQVIA's administration is flexible, occurring up to once monthly.
- 89.4% of patients reported adverse events.
- 60% of patients experienced adverse events related to HYQVIA.
- Serious adverse events related to HYQVIA occurred in three patients.
- Adverse events such as headaches, nausea, and fatigue were common.
- The mean infusion duration was long at 135.5 minutes per dose.
The long-term data from the ADVANCE-CIDP 3 clinical trial of HYQVIA provides robust evidence supporting its effectiveness and safety for treating chronic inflammatory demyelinating polyneuropathy (CIDP). This is important because HYQVIA is the first facilitated subcutaneous immunoglobulin (fSCIG) therapy approved for CIDP, offering a significant improvement in terms of administration and patient convenience. The ability to self-administer the treatment at home and the potential for monthly dosing reduce the burden on patients and healthcare systems. These factors suggest a substantial improvement in the quality of life for patients managing this chronic condition.
The trial results indicating a low annualized relapse rate and a favorable safety profile align with existing data on immunoglobulin therapies for CIDP. This bolsters confidence in the long-term use of HYQVIA, especially considering the standard of care for CIDP heavily relies on immunoglobulin therapies due to their immunomodulatory and anti-inflammatory effects. It’s worth noting that the study's extensive duration—up to 77 months—adds significant weight to its findings.
For retail investors, these results can indicate a stable and potentially growing market for HYQVIA as more patients and healthcare providers may prefer this treatment over traditional IVIG options due to its convenience and efficiency.
From a financial perspective, Takeda's positive long-term clinical trial results for HYQVIA are likely to enhance the value proposition of the company’s plasma-derived therapies portfolio. The market for immunoglobulin therapies is substantial and the introduction of a more convenient treatment option could lead to increased market share. The fact that nearly a quarter of all immunoglobulin therapy is used in treating CIDP underscores the significant revenue potential for HYQVIA.
Investors should consider the broader impact of these findings on Takeda's market positioning. HYQVIA’s approval by both the U.S. FDA and European Commission for CIDP positions it strongly in key markets. The clinical data’s emphasis on safety and tolerability, along with a very low rate of serious adverse events, helps mitigate risk concerns, which is a positive signal for long-term revenue stability.
In the short-term, awareness and adoption rates will be important to watch. Over the long-term, Takeda's ongoing commitment to research and development in this area—exemplified by the ADVANCE-CIDP clinical program—will likely support sustained growth and strengthen investor confidence.
HYQVIA’s positioning in the market benefits from its unique selling proposition as the first facilitated subcutaneous immunoglobulin (fSCIG) for CIDP. The ability to administer large immunoglobulin volumes subcutaneously, similar to intravenous administration, but with the convenience of less frequent dosing, sets it apart from competitors. The treatment’s design to be self-administered after adequate training also taps into the growing trend of home-based healthcare solutions, which have seen increased demand post-pandemic.
For retail investors, the next steps involve monitoring market penetration rates and uptake among patients and healthcare providers. Additionally, Takeda's strategic focus on rare neuroimmunological disorders highlights a niche but potentially lucrative market segment. Given the chronic nature of CIDP, continued patient adherence to HYQVIA could ensure sustained revenue streams.
Overall, the findings from this long-term study are likely to support Takeda’s competitive edge in the immunoglobulin therapy market, providing a solid basis for future growth and innovation.
- Favorable Findings from the Longest Public Clinical Study in CIDP Support HYQVIA as an Effective Long-Term Treatment Option for Maintaining Stable Disease Course in CIDP
- ADVANCE Clinical Program Reflects Takeda’s Commitment to Continued Research on the Role of Immunoglobulin (IG) Therapy for Patients with Rare Neuroimmunological Disorders
HYQVIA is the first and only facilitated subcutaneous immunoglobulin (fSCIG) for CIDP, approved earlier this year by the
“The long-term data from the ADVANCE-CIDP 3 clinical trial allow us to further characterize the safety, efficacy and tolerability profile of HYQVIA and reinforces its role as a long-term, up-to once monthly maintenance treatment for this complex, chronic condition,” said Kristina Allikmets, senior vice president and head of Research & Development for Takeda’s Plasma-Derived Therapies Business Unit. “These results reflect our continued commitment to bringing the benefits of our differentiated immunoglobulin therapies to patients with neuroimmunological disorders, and providing a range of effective treatment options that address the individual needs of a broad range of patients.”
The ADVANCE-CIDP 3 clinical trial is the longest extension study ever performed within context of a clinical trial in CIDP to date. The study, which enrolled 85 patients from the ADVANCE-CIDP 1 clinical trial, evaluated the safety, tolerability and immunogenicity of HYQVIA. The primary outcome measure was safety/tolerability and immunogenicity. The median duration of HYQVIA treatment was 33 months (0 to 77 months) with a cumulative overall follow-up time of 220 patient years. The findings were consistent with the known safety and tolerability profile of HYQVIA and no new safety concerns were observed.2 Key findings showed:
- The median monthly dose of HYQVIA across all patients was 64 (28.0 to 200.0) g/4 weeks.
-
The mean infusion duration per dose of HYQVIA was 135.5 minutes with
88.2% of doses administered every 4 weeks and92.3% of doses administered across two infusion sites. -
HYQVIA was well tolerated among the 3487 infusions administered; 3 (
0.1% ) infusions had a reduced infusion rate, were interrupted or stopped due to intolerability. -
Overall, adverse events (AEs) were reported in
89.4% of patients. AEs related to HYQVIA were reported in60% of patients. Most AEs were mild or moderate and self-limiting, and consistent with the established safety profile of HYQVIA. - The most common AEs per infusion (≥0.02 events per infusion) were headache, infusion site erythema, pyrexia, nausea, erythema, infusion site pruritis, fatigue and infusion site pain.
- Serious AEs possibly related to HYQVIA occurred in three patients (one event each): infection at the infusion site, exacerbation of migraine and fibromyalgia after infusion, and exacerbation of heart failure that resolved following treatment.
-
HYQVIA maintained stable disease course in patients with CIDP. Thirteen percent of patients with data available experienced a relapse during the entire observation period with an annualized relapse rate of
4.5% .
“Results of the ADVANCE-CIDP 3 study help provide additional confidence to those living with CIDP and their healthcare providers regarding the potential for extended maintenance of their condition with a facilitated subcutaneous immunoglobulin,” said Dr. Robert Hadden, MD, Consultant Neurologist, Neurology Department, King’s College Hospital,
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 the extremities.3 The role of IG therapy for CIDP has been well-established4 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.5 Nearly a quarter of all IG therapy is used in the treatment of CIDP.6
About HYQVIA®
HYQVIA® [Immune Globulin Infusion
About the ADVANCE-CIDP 3 Clinical Trial
ADVANCE-CIDP 3 was a long-term extension of ADVANCE-CIDP 1, a Phase 3, double-blind, randomized, placebo-controlled study.7 All patients entering ADVANCE-CIDP 3 received open-label HYQVIA and continued to receive the same dose and dosing regimen from ADVANCE-CIDP 1 (mean monthly dose equivalent of 1.1 g/kg).7 The primary objective was to analyze long-term safety, tolerability and immunogenicity. Efficacy was an exploratory outcome, including evaluation of CIDP relapse.
Further information about the ADVANCE-CIDP 3 clinical trial is available at ClinicalTrials.gov under study identifier NCT02955355.
HyQvia® (Human normal immunoglobulin) 100 mg/ml solution for infusion for subcutaneous use European 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
Indications: Replacement therapy in adults, children and adolescents (0-18 years) in: primary immunodeficiency syndromes (PID) 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 <4 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. Immunomodulatory therapy in adults, children and adolescents (0 to 18 years) in: chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy after stabilization with IVIg.
Dosage and administration: For subcutaneous use only. Therapy should be initiated and monitored under the supervision of a physician experienced in the treatment of immunodeficiency/CIDP. 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
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 and neutralizing 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.
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.
Interference with serological testing: After infusion of immunoglobulins, the transitory rise of the various passively transferred antibodies in the patient’s blood may result in misleading positive results in serological testing. Passive transmission of antibodies to erythrocyte´s surface antigens may interfere with some serological tests for red cell antibodies. Infusions of immunoglobulin products may lead to false positive readings in assays that depend on detection of β-D glucans for diagnosis of fungal infections.
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: The ability to drive and operate machines may be impaired by some adverse reactions e.g., dizziness associated with this medicinal product. Patients who experience adverse reactions during treatment should wait for these to resolve before driving or operating machines.
Undesirable effects: Very common (≥1/10 patients): Headache, Blood pressure increased and Hypertension, Nauseam Diarrhoea, Vomiting, Arthralgia, Local reactions (Infusion site discomfort, Infusion site pain, Injection site pain, Puncture site pain and Tenderness; infusion site erythema and Injection site erythema; Infusion site oedema, Injection site oedema, infusion site swelling, Injection site swelling and Swelling (local), Feeling hot, Asthenia, Fatigue, Lethargy and Malaise.
Common (≥1/100, <1/10 patients): Migraine, Tremor, Paraesthesia, Sinus tachycardia and Tachycardia, Hypotension, Dyspnoea, Abdominal distension, Erythema, Pruritus, Rash, Rash erythematous, Rash macular, Rash maculo-papular and Rash popular Urticaria, Myalgia, Limb discomfort and Pain in extremity, Back pain, Joint stiffness, Musculoskeletal chest pain, Groin pain, Hemosiderinuria, Infusion related reaction, Infusion site bruising, Injection site bruising, Infusion site haematoma, Injection site haematoma, Infusion site haemorrhage and Vessel puncture site bruise, Infusion site reaction, Injection site reaction and Puncture site reaction, Infusion site mass, Injection site mass and Infusion site nodule, Infusion site discoloration, Infusion site rash and Injection site rash, Infusion site induration and Injection site induration, Infusion site warmth, Infusion site paraesthesia and Injection site paraesthesia, Infusion site inflammation, Chills, Oedema, Oedema peripheral and Swelling (systemic), Localised oedema, Peripheral swelling and Skin oedema, Gravitational oedema, Oedema genital, Scrotal swelling and Vulvovaginal swelling, Hyperhidrosis, Coombs direct test positive and Coombs test positive.
Uncommon (≥ 1/1 000 to < 1/100): Cerebrovascular accident and Ischaemic stroke, Burning sensations.
Other undesirable effects (rare or unknown frequency): Meningitis aseptic, Hypersensitivity, Direct Coombs’ test positive, Infusion site leakage, Influenza-like illness.
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
PI approval code: PI-02941
Date of preparation: June 2024.
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.
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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
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References
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1 European Medicines Agency. HyQvia 100 mg/mL solution for infusion for subcutaneous use Summary of Product Characteristics. Available at https://www.ema.europa.eu/en/documents/product-information/hyqvia-epar-product-information_en.pdf.
2 Hadden R et al. Hyaluronidase-facilitated subcutaneous immunoglobulin
3 Dalakas MC; Medscape. Advances in the diagnosis, pathogenesis and treatment of CIDP. Nat Rev Neurol. 2011;7(9):507-517.
4 Eftimov F, et al. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2013;(12):CD001797.
5 Van den Bergh PYK, et al. 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 [published correction appears in J Perpher Nerv System. 2022 Mar;27(1):94].
6 Adivo 2020 data (incl. US,
7 ClinicalTrials.gov. Long-Term Tolerability and Safety of HYQVIA/HyQvia in CIDP. July 24, 2023. Last accessed May 2024 from https://clinicaltrials.gov/study/NCT0295535
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Jun Saito
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Source: Takeda Pharmaceutical Company Limited
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