Cutaquig FDA Approved for Treatment of Children with Primary Humoral Immunodeficiency

According to a recent article from Pharmacy Times, Octapharma’s cutaquig has been approved by the FDA to treat patients who are 2 years of age or older with primary humoral immunodeficiency (PI).

Primary Immunodeficiency Diseases (PI)

Primary immunodeficiency (PI) diseases are inherited immune system disorders, caused by a mutated gene that prevents the body’s immune system from fighting infection effectively. Because they are genetic in nature, these immune system disorders are commonly passed from one generation to the next, but not always. There are many instances where a person is born with a mutated gene, and is the only one in the family to have a primary immunodeficiency.

PIs can be difficult to diagnose because there are more than 200 known forms of this particular immune system disorder, and symptoms vary. For example, a person with chronic granulomatous disease (CGD) may have difficulty fighting fungal and bacterial infections whereas a person with common variable immunodeficiency disease (CVID) is prone to infection from flu strains, pneumonia, and streptococcus pneumonia. The bottom line of any PI disorder is that part of the immune system is absent or not functioning properly; thus, patients are susceptible to different infections throughout their lives.

One in every 2,000 people are affected by an autoimmune disease, including more than 500,000 people in the United States, but researchers say the numbers may be much higher.

More Flexible Treatment Options

The FDA approval of cutaquig is giving families and their physicians more treatment options. Based on a patient’s response to the infusions, the amount of infusions can be higher or lower, allowing more flexible options for patients. Physicians can also adjust the dose levels depending on a patient’s wants and needs. Cutaquig is allowing a level of convenience to a patient’s lifestyle that has not been seen before for patients with PI.

Clinical Trials

The FDA approved cutaquig based off of 2 clinical trials. The trials, which looked to assess cutaquig’s ability to prevent serious bacterial infections,  consisted of 75 people with PI. Those in the trial received infusions weekly with cutaquig during a 12 week wash-in/wash-out period, followed by a 12 month efficacy period. The results of the trials were that no bacterial infections were reported.

Since cutaquig is an immune globulin product, there are possible complications. Thrombosis (when blood clots block veins and arteries) may occur, especially if a patient has one of the following risk factors: advanced age, cardiovascular issues, estrogen use, arterial or venous thrombosis past history, hypercoagulable conditions, hyperviscosity, long periods of immobilization, and indwelling vascular catheters. If a patient is at risk for thrombosis, the solution should be administered at low doses and infusions rates. Furthermore, patients should be monitored closely for any signs or symptoms of thrombosis by their physician.

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