Now FDA-Approved: Cutaquig for Pediatric Patients with PI 

It is extremely important to get the right medications into the hands of patients who need them. Recently, the FDA helped do this for pediatric patients (aged 2+) with primary humoral immunodeficiency (PI). According to a news release from human protein product manufacturer Octapharma USA (“Octapharma”), the FDA recently approved cutaquig (16.5% solution) for use within this patient population. Cutaquig is already approved for use in adults with primary immunodeficiency.

Cutaquig

To begin, let’s take a look at exactly what cutaquig is. According to Octapharma, cutaquig is a:

Liquid, ready-to-use 16.5% immune globulin solution for subcutaneous infusion (SCIg)…available in 6 mL (1g), 12 mL (2g), 24 mL (4g), and 48 mL (8g) convenient, ready-to-use vial sizes for flexibility in delivering SCIg to patients with varied needs.

The approval comes following data from two separate clinical trials. Altogether, 75 patients enrolled, with about a 50/50 split on adults and children. Researchers found that the therapy was effective in preventing serious or life-threatening bacterial infections from occurring. This is extremely important for patients with PI, as this condition stops the immune system from effectively fighting infections. Thus, having the extra support from cutaquig is extremely beneficial.

Cutaquig is also more convenient than other therapeutic options. For example, patients can personalize their treatment schedules: more frequent lower doses or less frequent higher doses.

While cutaquig is relatively safe and well-tolerated, physicians prescribing the treatment to patients should keep an eye out for thrombosis.

Primary Immunodeficiency (PI)

There are over 350 rare and chronic inherited immune system disorders under the umbrella of primary immunodeficiency (PI). For example, common variable immunodeficiency disease (CVID), severe combined immunodeficiency disease (SCID), and chronic granulomatous disease (CGD) are all subsets of PI. In each case, mutated genes stop the body’s immune system from functioning properly. For these reasons, many people with PI have extreme difficulty fighting infections. The infections also differ based on subtype. For example, those with CGD may struggle with fungal or bacterial infections. Alternately, people with CVID are more prone to streptococcus pneumonia. Regardless, those with PI have difficulty with full immune function.

Because there are many subtypes, symptoms also vary. However, some potential symptoms associated with PI include:

  • Frequent, recurrent, and difficult-to-treat infections
  • Anemia (low red blood cell count)
  • Thrombocytopenia (low platelet count)
  • Delayed growth or development
  • Organ inflammation and infection
  • Appetite loss
  • Nausea and vomiting
  • Abdominal pain or cramping
  • Diarrhea
  • Autoimmune disorders (lupus, type 1 diabetes)
Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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