Takeda Reports Positive Full Data Set from Phase 3 Trial of Hyqvia as a Maintenance Therapy for CIDP

Rare Daily Staff

Takeda reported positive full results from the pivotal phase 3 ADVANCE-CIDP 1 clinical trial studying Hyqvia as maintenance therapy in adult patients with the rare immune disorder chronic inflammatory demyelinating polyneuropathy.

Results showed a clinically significant reduction in relapse rate of 9.7 percent with Hyqvia vs a 31.4 percent placebo and other analysis showed delayed time to relapse with Hyqvia vs. placebo. The company also observed favorable data across other endpoints from the study and favorable tolerability. The findings were presented at the 2023 Peripheral Nerve Society Annual Meeting in Copenhagen and published in the Journal of the Peripheral Nervous System.

Chronic inflammatory demyelinating polyneuropathy (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. The role of immunoglobulin (IG) therapy for this slowly progressing or relapsing disease has been well-established. IG is considered a standard of care for this complex and heterogeneous disease due to its broad immunomodulatory and anti-inflammatory effects. However, the high volume and frequency of treatment required to effectively manage this disease means that treatment can be a burden for patients and their health care providers.

Hyqvia contains recombinant human hyaluronidase and immunoglobulins (Ig) and is approved by the European Medicines Agency 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 less than 4 g/L. It is also approved in the United States to treat adults and children 2 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).

Hyqvia is currently under regulatory review in the United States and European Union for use as a maintenance therapy in adult patients with stable CIDP.

ADVANCE-CIDP 1 is a phase 3, prospective, randomized, double-blind, multicenter, placebo-controlled study in which adults with stable CIDP on intravenous immunoglobulin (IVIG) were randomized 1:1 to be switched to Hyqvia or placebo and received their assigned treatment for six months or until relapse or study withdrawal.

In addition to showing a clinically significant reduction in relapse rate compared to placebo, Hyqvia showed a lower probability of functional worsening rates versus placebo (37.5 percent vs 54.4 percent). Patients receiving Hyqvia experienced longer time to relapse compared to those receiving placebo and change in R-ODS centile scores were lower in the Hyqvia group than the placebo group.

The safety profile of Hyqvia in the ADVANCE-CIDP 1 trial was generally consistent with the existing European Union summary of product characteristics. Infusion characteristics were well-matched between Hyqvia and placebo groups, with less than 1 percent of all infusions affected by intolerability and/or adverse events.

In the Hyqvia treatment arm, the most common causally related local adverse events (less than 5 percent of patients) included injection and infusion site pain and erythema, and infusion site edema and pruritis. The most common causally related systemic adverse events (less than 5 percent of patients) included headache, nausea, fatigue, and pruritus.

“For patients with CIDP who need immunoglobulin treatment, the ADVANCE-CIDP 1 study results are encouraging,” said Robert Hadden, consultant neurologist with the Department of Neurology at King’s College Hospital in London. “If approved as a maintenance therapy for CIDP, this treatment may combine the ability to have subcutaneous treatment at home with less frequent infusions.”