US regulators have cleared CSL Behring’s Hizentra for the treatment of chronic inflammatory demyelinating polyneuropathy (CIDP), a rare autoimmune disorder affecting the peripheral nerves that can cause permanent nerve damage.
In CIDP, the myelin sheath, the protective covering of the nerves, is damaged, which may result in numbness or tingling, muscle weakness, fatigue, and other symptoms. Effects can worsen over time, significantly limiting activity and decreasing quality of life. Around 30 percent of CIDP patients will progress to wheelchair dependence if not treated.
The approval of Hizentra gives patients the option of accessing the first and only subcutaneous immunoglobulin (SCIg) as maintenance therapy to prevent relapse of neuromuscular disability and impairment.
Clearance follows data from the Phase III PATH trial, the largest controlled clinical study in CIDP patients to date, which showed that the percentage of patients experiencing relapse or withdrawal for any other reason during SCIg treatment was significantly lower with Hizentra - 38.6 percent on low-dose and 32.8 percent on high-dose - than with placebo (63.2 percent).
The PATH study also demonstrated that patients on Hizentra reported fewer systemic adverse reactions per infusion compared to intravenous therapy (2.7 percent versus 9.8 percent, respectively). In fact, 93 percent of the 4,225 total Hizentra infusions were free of any adverse reactions, the firm noted.
"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.
"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."