Primary Endpoints Met in Phase 3 of Hizentra for Polyneuropathy

Mathew Shanley

New data published earlier this week in The Lancet Neurology concluded that Hizentra (Immune Globulin Subcutaneous [Human]) is safe and effective in treating chronic inflammatory demyelinating polyneuropathy (CIDP) based on results from the Phase 3 PATH study (Polyneuropathy And Treatment with Hizentra) meeting its primary endpoints.

172 patients with CIDP were randomized in the study; 33% (n=57) received placebo, 33% (n=57) received low doses (0.2 g/kg weekly) of Hizentra, and 34% (n=58) received high doses (0.4 k/kg weekly) of Hizentra. Over a 24-week period, 39% (n=22) of patients in the low-dose cohort and 33% (n=19) on high-dose Hizentra experienced a CIDP relapse or withdrew from the study in comparison to 63% (n=35) of patients on placebo.

CIDP is a disorder in the autoimmune system, characterized by the attack of myelin sheath in the peripheral nervous system. Common symptoms include numbness and tingling throughout the body and muscle weakness and frequent fatigue. Per the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM), an estimated 30% of CIDP patients will progress to dependence on wheelchairs if not treated properly and quickly.

In September, the U.S. Food and Drug Administration’s (FDA) approved the intravenous administration of the immunoglobulin (Privigen) for the treatment of CIPD. The PATH study tested subcutaneous administration of the immunoglobulin.

"Subcutaneous administration of immunoglobulin has gained popularity as an alternative route of administration but has never been rigorously examined for the treatment of CIDP until the PATH study," said Prof. Dr. Ivo N. van Schaik, principal investigator, lead author of The Lancet Neurology publication in a press release, and professor of neurology at the University of Amsterdam's Faculty of Medicine (AMC-UvA). "In this groundbreaking study, Hizentra maintained stable disease and prevented relapse, suggesting that subcutaneous immunoglobulin may be used as an alternative maintenance therapy to intravenous immunoglobulin in CIDP patients."

Hizentra is currently approved as a replacement therapy for patients aged 2 years and older with primary humoral immunodeficiency (PI). CSI Behring is currently waiting responses on its applications in both the U.S. and Europe for a CIDP indication for the subcutaneously administered immunoglobulin (SCIG).

Adverse events (AEs) occurred in 58% (n=33) of the low-dose group and 52% (n=30) of the high-dose group, and most consisted of local reactions at the infusion site. 95% of local reactions were mild and 5% were moderate, and only 11 total serious AEs were reported, 1 of which led to discontinuation of treatment.

"CSL Behring is committed to advancing innovative medical research to unlock the promise of immunoglobulins for the treatment of patients with disabling neurologic conditions, including CIDP. The PATH study underscores this commitment," said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D director of CSL Limited. "If Hizentra is approved as a maintenance treatment for CIDP by regulatory authorities, we will be able to offer patients a flexible treatment option that allows them the freedom to self-administer immunoglobulin at a convenient place and time."

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