This morning, it was announced that the U.S. Food and Drug Administration (FDA) has approved CSL Behring’s maintenance therapy for the treatment of patients with chronic inflammatory demyelinating polyneuropathy (CIDP).
Immune Globlulin Subcutaneous [Human] 20% Liquid (Hizentra) is the first and only subcutaneous immunoglobin (SCIg) approved for the rare disease, and was developed to prevent relapse of neuromuscular disability and impairment, which are two common symptoms of patients with the condition.
It was previously greenlighted by the FDA for the treatment of primary immunodeficiency (PI) and adults and pediatric patients, but now offers patients with CIDP a convenient treatment option with established efficacy, as exhibited in the largest controlled study in CIDP patients to date.
The Phase 3 PATH (Polyneuropathy And Treatment with Hizentra) study demonstrated that the percentage of patients with CIDP experiencing relapse or withdrawal for any other reason during SCIg treatment was significantly lower with the drug than with placebo. The study results
were published in The Lancet Neurology
in November 2017.
172 patients with CIDP were randomized in the study; 33% received low doses (0.2 g/kg weekly) of Immune Globulin Subcutaenous [Human] 20% Liquid, and 34% (n=58) received high doses. 33% (n=57) received placebo. Over a 24-week period, 39% (n=22) of patients in the low-dose cohort and 33% (n=19) in the high-dose cohort experienced a CIDP relapse or withdrew from the study in comparison to 63% (n=35) of patients on placebo.
"This new FDA approval for Hizentra marks a pivotal milestone for patients struggling with the disabling neurological effects of CIDP," said Dr. Andrew Cuthbertson, Chief Scientific Officer and R&D Director, CSL Limited in a press release
. "As the first and only subcutaneous immunoglobulin therapy approved to treat CIDP, and studied in the largest controlled clinical trial for CIDP, Hizentra offers patients a more convenient treatment option with proven efficacy and the flexibility and freedom to self-infuse at home. CSL Behring is deeply committed to delivering innovative therapies to patients with rare and serious diseases like CIDP, and today we are proud to now offer a portfolio of immunoglobulin therapies for CIDP."
The study also demonstrated that patients on Immune Globlulin Subcutaneous [Human] 20% Liquid reported fewer systemic adverse reactions (ARs) per infusion compared to IVIg treatment (2.7% versus 9.8%, respectively).
"Despite available treatment options, many CIDP patients continue to struggle with daily disease and lifestyle challenges, making continued research and innovation critical," said Lisa Butler, executive director of the GBS|CIDP Foundation International. "The approval of Hizentra offers patients who were once burdened by traveling to the infusion center or hospital the flexibility to self-administer their treatment at a time, place, and on a schedule that's convenient for them."
In the PATH study, adverse events (AEs) occurred in 58% (n=33) of patients in the low-dose group and 52% (n=30) of patients in the high-dose group. Most consisted of local reactions at the infusion site.
"As a practicing neurologist treating patients with CIDP, I am excited to have a safe and effective subcutaneous treatment option to offer my patients who are interested in more treatment flexibility and control in their busy lives," said Dr. Todd Levine, Phoenix Neurological Associates.
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