RARE Daily

FDA Approves GC Biopharma’s Alyglo for Rare Immunodeficiencies

December 18, 2023

Rare Daily Staff

The U.S. Food and Drug Administration approved GC Biopharma’s Alyglo for the treatment of adult patients aged 17 years and older with primary humoral immunodeficiency.

Primary humoral immunodeficiency (PI) refers to a group of disorders where the body’s ability to produce antibodies is impaired, affecting the immune system’s ability to fight infections. In PI, there’s a deficiency or absence of B cells or plasma cells, which are key in producing antibodies, particularly immunoglobulin G. This leads to increased susceptibility to infection, especially bacterial infections affecting the respiratory and gastrointestinal tracts.

People with PI might experience frequent infections, some of which can be severe or chronic. Treatment often involves immunoglobulin replacement therapy to boost the immune system. Vaccinations and antibiotics may also be used to prevent and treat infections. The exact type and severity of PI can vary widely among individuals.

Alyglo is a liquid solution containing 10 percent immunoglobulin G (100 mg/mL) for intravenous infusion, manufactured from pooled human plasma from U.S. donors. The manufacturing process includes three steps to reduce the risk of virus transmission.

The Alyglo pivotal phase 3 clinical study followed FDA guidance for the treatment of patients with PI. The clinical trial was a prospective, open-label, single-arm, historically controlled, multicenter phase 3 study to assess the efficacy and safety of Alyglo in patients with a confirmed diagnosis of PI. The studies were conducted in the United States and Canada.

The study met its primary efficacy endpoint with 0.03 acute serious bacterial infections (aSBIs) per patient-year, less than the FDA efficacy requirement of less than one aSBI per patient-year.

The proportion of infusions with temporally associated adverse events occurring during or within 72 hours after infusion was 0.22, less than the FDA-required prespecified end point of less than 0.40.

“Alyglo will significantly impact clinical practice in the U.S. due to its strong safety and efficacy profile,” said Elena Perez, allergy Immunologist from Allergy Associates of the Palm Beaches, and lead study investigator. “It is an important treatment option for primary immune-deficient patients. “

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