The U.S. Food and Drug Administration (FDA) has approved Privigen [Immune Globulin Intravenous (Human), 10% Liquid] for treating adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability.
CIPD is a rare autoimmune disorder that attacks myelin sheath in the peripheral nervous system. Symptoms vary from numbness or tingling to muscle weakness and fatigue. Approximately 30% of CIDP patients will eventually require a wheelchair if not managed properly. The neurological condition affects about 40,000 people in the U.S.
The approval is based on 2 Phase 3 studies, the P
reatment with H
izentra (PATH) study and the Pr
mpact on M
obility and A
utonomy (PRIMA) study.
In the PATH study, 73% of the 207 patients receiving Privigen responded to treatment [as measured by their adjusted score on the Inflammatory Neuropathy Cause and Treatment (INCAT) scale] while in the PRIMA study, 61% of the 28 patients responded to treatment.
In a news release, Mazen M. Dimachkie, Professor and Director of Neuromuscular Division, Executive Vice Chairman, Department of Neurology at the University of Kansas Medical Center and an investigator in the PATH study said, "It is a priority in the care of CIDP patients to provide therapies that improve and maintain strength and function while at the same preventing relapses and minimizing side effects. However, current treatments do not work for all CIDP patient." Dr. Dimachkie added, "Privigen's approval by the FDA for the treatment of CIDP means that people with CIDP and their treating physicians have gained another treatment option that is safe and effective in helping improve strength and motor function, while potentially delaying disease relapse."
"People living with CIDP can experience a progression of their disease, which may result in tingling, muscle weakness, fatigue and other symptoms that limit their daily activities and decrease their quality of life," said Lisa Butler, executive director of the GBS|CIDP Foundation International. "The approval of this intravenous immunoglobulin to improve disability represents an important treatment advance for the patients, caregivers and families who are struggling with CIDP. We are grateful for CSL Behring's efforts in this area and their commitment to advancing patient care for the CIDP."
Privigen is a IVIG solution stabilized with proline that is also approved for primary humoral immunodeficiency (PI) and chronic immune thrombocytopenic purpura (ITP).
The drug comes with a boxed warning regarding its use may put patients at risk for thrombosis, renal dysfunction, and acute renal failure.
For more from the FDA, including applications, designations and approvals, follow Rare Disease Report on Facebook
. For breaking news sent straight to your inbox, sign up for the RDR e-newsletter