FDA Approves Octapharma’s Cutaquig for Primary Immunodeficiency in Kids

The U.S Food and Drug Administration approved Octapharma USA’s cutaquig for the treatment of pediatric patients ages 2 and older with primary humoral immunodeficiency.

The FDA previously approved cutaquig for adults with PI.

Primary immunedeficiencies (PI) are a group of more than 300 disorders (many of which are rare) in which part of the body’s immune system is missing or does not function properly. When any part of a person’s immune system is absent or dysfunctional, the individuals are susceptible to infections, and it may take longer to recover from infections. Some rare primary immunodeficiencies include: Wiskott-Aldrich Syndrome, DiGeorge Syndrome, Hyper IgE Syndrome, Ataxia-Telangiectasia, and many more.

Cutaquig is a 16.5 percent immune globulin solution for subcutaneous infusion indicated for treatment of primary humoral immunodeficiency (PI) in adults and pediatric patients 2 years of age and older.

“The FDA approval provides physicians and families with more treatment options for patients with primary immune disorders, which weaken the immune system and can allow infections and other health issues to occur more easily,” said Roger Kobayashi, clinical professor at the UCLA School of Medicine and national consultant for the Immune Deficiency Foundation. “The FDA approval also provides more flexible options by permitting more frequent or less frequent infusions, which can be advantageous based on a patient’s pharmacokinetic and clinical response.”

Patients and providers have the flexibility to administer cutaquig at a lower dose more frequently or at a larger dose less frequently if desired. Patients who prefer less frequent injections may have the option of receiving therapy every other week. At the same time, physicians can prescribe daily dosing if patients respond better to more frequent therapy.

“Cutaquig provides enhanced convenience for a wider group of patients who want to customize therapy with their prescriber to best match patient lifestyle needs,” said Octapharma USA President Flemming Nielsen.

The FDA approval of cutaquig is based on the results of two clinical trials, which observed 75 PI patients, 37 adults and 38 pediatric patients between ages 2 and 17. The patients received weekly infusions with cutaquig during a 12-week wash-in/wash-out period followed by a 12-month efficacy period.

The main objective of the research was to assess the efficacy of cutaquig in preventing serious bacterial infections, defined as bacteremia/sepsis, bacterial meningitis, osteomyelitis/septic arthritis, bacterial pneumonia, and visceral abscess. No serious bacterial infections were reported.

Photo: Flemming Nielsen, president of Octapharma USA  

Author: Rare Daily Staff