Takeda Hyqvia gets European approval for maintenance therapy in patients with chronic inflammatory demyelinating polyneuropathy

Takeda announced that the European Commission (EC) approved Hyqvia [Immune Globulin Infusion 10% (Human) with Recombinant Human Hyaluronidase] as maintenance therapy in patients of all ages with chronic inflammatory demyelinating polyneuropathy (CIDP) after stabilization with intravenous immunoglobulin therapy (IVIG). Takeda previously announced a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) on December 15, 2023 and approval as a maintenance therapy for adults with CIDP by the US Food and Drug Administration on January 16, 2024.

As the first and only facilitated subcutaneous immunoglobulin (fSCIG) for CIDP, Hyqvia offers the potential for patients to infuse up to once monthly (every two, three or four weeks), as the hyaluronidase component facilitates the dispersion and absorption of large immunoglobulin (IG) volumes in the subcutaneous space between the skin and the muscle. Hyqvia can be administered by a healthcare professional or self-administered in the comfort of a patient’s own home after appropriate training.

“Following the FDA approval of the Hyqvia CIDP indication in January 2024, the EC’s approval of Hyqvia for CIDP is a critical step towards giving people in the EU living with CIDP access to a maintenance treatment with proven efficacy that can be administered up to once monthly, at-home or in-office” said Kristina Allikmets, senior vice present and head of research & development for Takeda’s Plasma-Derived Therapies Business Unit. “This expanded indication for HYQVIA also reflects Takeda’s commitment to bring the benefits of our immunoglobulin therapies to people with neuroimmunological disorders and provide treatment options that have the potential to positively impact their lives and elevate the standard of care.”

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. The role of IG 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.

This approval is based on data from the pivotal phase 3 ADVANCE-CIDP 1 trial, which was a multicenter, placebo-controlled, double-blinded study that evaluated the efficacy and safety of Hyqvia as a maintenance therapy to prevent relapse in patients with CIDP. The global study included 132 adults with a confirmed diagnosis of CIDP who had remained on a stable dosing regimen of IVIG therapy for at least three months prior to screening. Results showed a clinically significant reduction in CIDP relapse rate with Hyqvia versus placebo 15.5% (95% CI: 8.36, 26.84) in the Hyqvia and 31.7% (95% CI: 21.96, 43.39) in the placebo groups. The treatment difference was -16.2 (95% CI: -29.92, -1.27), favouring Hyqvia over placebo.

While adverse events (AEs) were more frequent with Hyqvia (79.0% of patients) than placebo (57.1%), severe (1.6% vs 8.6%) and serious AEs (3.2% vs 7.1%) were less common. The majority of AEs were mild or moderate, local, did not require suspension of infusions, and resolved without sequelae. The most common (reported in >5% of patients) causally related AEs included headache and nausea, as well as local AEs including infusion site pain, erythema, pruritis, and edema. 7 Overall, the safety profile observed in the ADVANCE-CIDP 1 trial was generally consistent with the existing EU Summary of Product Characteristics (SmPC).

The centralized marketing authorization for Hyqvia in CIDP is valid in all EU member states as well as in Iceland, Liechtenstein, Norway and Northern Ireland. Hyqvia first received approval from the EC for the treatment of primary immunodeficiency (PID) in 2013 as well as secondary immunodeficiency (SID) in 2020.

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. In addition, it is approved by the EMA as maintenance therapy in adults, children and adolescents (0-18 years) with chronic inflammatory demyelinating polyneuropathy (CIDP) after stabilization with intravenous immunoglobulin therapy (IVIG). In the United States it is approved to treat adults and children two years of age and older with PI as a well as a maintenance therapy for adult patients with CIDP. 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).

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 ?2 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.

Takeda is focused on creating better health for people and a brighter future for the world. The company 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.

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