The FDA has just provided Orphan Drug Designation to Hizentra for the treatment of adults diagnosed with chronic inflammatory demyelinating polyneuropathy (CIPD). This is a maintenance therapy which works to prevent relapse.
CIPD impacts the peripheral nerves and can cause neuromuscular disability. Patients may experience fatigue, muscle weakness, numbness, and tingling as well as other symptoms. Onset may occur at any age and the effects of the disease can worsen as time goes on. Around 30% of patients will be wheelchair dependent if they don’t receive treatment.
Hizentra is the most prescribed SCIg in the world. It has a record of its safety, tolerability, and efficacy. It’s first approval came in 2010 for primary immunodeficiency. Its has now been provided to 6.7 million patients in 60 countries.
It was approved in 2018 by the FDA for adult CIDP patients, making it the first SCIg approved for this patient population. The approval was the result of the successful Phase III PATH trial where the treated patients relapsed less than those who didn’t receive the therapy. The Hizentra group also maintained their body strength better than the placebo group. This investigation was the largest CIDP clinical trial in history.
This new designation gives CSL Behring 7 years of marketing exclusivity of the therapy in the United States. It also will help provide incentives for other companies to invest in the continued research of this treatment.
You can read more about this therapy here.