CSL Behring announced Hizentra has received orphan-drug exclusivity from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with chronic inflammatory demyelinating polyneuropathy (CIDP) as maintenance therapy to prevent relapse of neuromuscular disability and impairment. CIDP is a rare autoimmune disorder that affects the peripheral nerves and has the potential to cause significant disability.
Hizentra was approved by the FDA in March 2018 for the treatment of adults with CIDP to prevent relapse of neuromuscular disability and impairment. It is the first and only 20 percent subcutaneous immunoglobulin (SCIg) approved for this indication. The approval was based on data from the Phase III PATH (Polyneuropathy And Treatment with Hizentra) study, the largest controlled clinical study in CIDP patients to date. In the PATH trial, patients taking Hizentra relapsed or withdrew less often than those taking placebo. Patients in the study also maintained their grip strength as well as upper and lower body strength.
The FDA's decision regarding the orphan drug status of Hizentra provides CSL Behring a seven- year period of U.S. marketing exclusivity for Hizentra in the maintenance treatment of CIDP with SCIg. The FDA Office of Orphan Products Development grants orphan designation for novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. and qualifies incentives for companies that invest in research and development of these medications.
Subscribe to our e-Newsletters
Stay up to date with the latest news, articles, and events. Plus, get special offers
from American Pharmaceutical Review – all delivered right to your inbox! Sign up now!
"Orphan drug exclusivity is a significant milestone for the CSL Behring team committed to delivering Hizentra and improving the lives of patients diagnosed with CIDP," said Bob Lojewski, Senior Vice President and General Manager, North America, CSL Behring. "We are proud to be the only company to offer an innovative portfolio of subcutaneous and intravenous immunoglobulin therapies for CIDP. Driven by our promise, we will continue our work to fulfill the needs of patients with serious conditions like CIDP."
CIDP is a rare autoimmune disorder that affects the peripheral nerves (those outside the brain and spinal cord) and damages the protective covering of the nerves known as the myelin sheath. This may result in numbness or tingling, muscle weakness, fatigue, and other symptoms. CIDP effects can worsen over time, leading to significant activity limitations and a decreased quality of life. CIDP can occur at any age and is more common in men than in women. The GBS/CIDP Foundation estimates that approximately 30 percent of CIDP patients will progress to wheelchair dependence if not treated. The Foundation also estimates that the incidence of CIDP in the U.S. is as high as two in 100,000 people each year, with the accumulation of cases over time resulting in prevalence as high as nine in 100,000 in some areas.