New Rare Disease Treatments Approved by Food and Drug Administration

The therapies will treat auto-inflammatory diseases, some of which had no prior approved therapies

New Rare Disease Treatments Approved by Food and Drug Administration
Image: Pixabay
September 28, 2016

The Food and Drug Administration has approved three new therapies for a medication known as Ilaris.

The therapies are intended to treat rare and serious auto-inflammatory diseases in both adult and pediatric patients, including two diseases for which there were no previously approved therapies. The diseases to be treated include:

  • Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS)
  • Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD)
  • Familial Mediterranean Fever (FMF)

Each of these is a hereditary disease characterized by periodic fever and inflammation as well as intense muscle pain. TRAPS and HDS/MKD had no prior approved treatment therapies.

"For the first time, patients with TRAPS and HIDS/MKD, two painful and life altering diseases, have access to a treatment that may help improve their quality of life," said Badrul Chowdhury, M.D., Ph.D, director of the Division of Pulmonary, Allergy and Rheumatology Products in FDA's Center for Drug Evaluation and Research.

Prior to the agency's approval of the medication for treating these syndromes, it had been approved to treat another periodic fever syndrome known as Cryopyrin-Associated Periodic Syndromes (CAPS) as well as for active systemic juvenile idiopathic arthritis.

Healthcare professionals should examine the label's prescribing information for details regarding approved uses of the drug.

The FDA's approval of the drug for the new therapies was based on clinical studies that included data on its safety, efficacy, and pharmacokinetics.

The most common reactions to these therapies are reactions at the injection site and increased susceptibility to colds. However, Ilaris can cause additional serious side effects, including the increased risk of serious infections, the decreased ability to fight infections, and allergic reactions.

Patients who experience any of the following symptoms of an allergic reaction should contact their healthcare provider: rash, itching and hives, difficulty breathing or swallowing, dizziness, or feeling faint. Patients who receive Ilaris should not receive live vaccines. Finally, anyone allergic to canakinumab or any of the ingredients in Ilaris should not receive the drug.