FDA Approves CSL Behring’s Hizentra for Patients with Chronic Inflammatory Condition that Damages Nerves

Rare Daily Staff

The U.S. Food and Drug Administration approved CSL Behring’s Hizentra, the first and only subcutaneous immunoglobulin for the treatment of chronic inflammatory demyelinating polyneuropathy, a rare autoimmune disorder that affects the nerves outside the brain and spinal cord.

In CIDP, the protective covering of the nerves known as the myelin sheath becomes damaged, which may result in numbness or tingling, muscle weakness, fatigue, and other symptoms. The effects can worsen over time, limiting activities and decreasing quality of life. Approximately 30 percent of CIDP patients will progress to wheelchair dependence if not treated.

Hizentra is a maintenance therapy intended to prevent relapse of neuromuscular disability and impairment. Hizentra is currently approved in over 51 countries and approved to treat certain immune deficiencies. 

The approval was based on data from the phase 3 study, which is the largest controlled clinical study in CIDP patients to date. The study showed that the percentage of patients experiencing CIDP relapse or withdrawal for any other reason during treatment was significantly lower with Hizentra than with placebo. The study also showed that patients on Hizentra reported fewer systemic adverse reactions per infusion compared to intravenous immunoglobulin treatment.

“This new FDA approval for Hizentra marks a pivotal milestone for patients struggling with the disabling neurological effects of CIDP,” said Andrew Cuthbertson, chief scientific officer and R&D Director for CSL Limited. “Hizentra offers patients a more convenient treatment option with proven efficacy and the flexibility and freedom to self-infuse at home.”

March 19, 2018
Photo: Andrew Cuthbertson, chief scientific officer of CSL Behring