New Products, Rare Diseases

FDA OKs Hizentra for rare autoimmune disease CIDP

Just one day after getting cleared to sell its new rare disease drug Hizentra in Europe, Pennsylvania drugmaker CSL Behring got the green light from US regulators to market the drug in the states.

The med is a maintenance therapy for a rare autoimmune disease called chronic inflammatory demyelinating polyneuropathy, or CIDP. The condition, also known as Vidaurri’s disease, is believed to be caused by misguided immune cells attacking the nerves of the body instead of protecting the body from infection. As a result, the affected nerves fail to respond, or respond only weakly, and on occasion, inordinately, causing numbing, tingling, pain, progressive muscle weakness, loss of deep tendon reflexes, fatigue, and abnormal sensations. Patients are generally treated with intravenous immunoglobulin.

CSL’s new drug Hizentra is a subcutaneous immunoglobulin, which can be used at home instead of requiring patients to visit infusion centers or hospitals. The drug was OK’ed as a treatment to prevent relapse of neuromuscular disability and impairment.

“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,” said Andrew Cuthbertson, CSO and R&D director at CSL Limited, in a statement.

The approval was based on data from the Phase III trial the company calls PATH (Polyneuropathy and treatment with Hizentra) study, which it says is the largest controlled clinical study in CIDP patients to date. The trial showed patients on Hizentra fared significantly better than the placebo group. The study showed 63.2% of patients on the placebo experienced CIPD relapse or withdrawal during treatment, while only 38.6% of patients on the low-dose of Hizentra experienced the same. On the high dose, it was only 32.8% of patients.

In the trial, patients on Hizentra also reported fewer systemic adverse reactions per infusion compared to IVIg treatment.

Hizentra is currently approved in 51 countries for the treatment of certain immune deficiencies. Yesterday, the drug was OK’ed by the European Commission for the treatment of CIDP.


Image: CSL CSO and R&D director Andrew Cuthbertson. CSL Behring


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