FDA Approves New Indication for Gammagard Liquid

The FDA has approved Takeda’s Gammagard Liquid as an intravenous immunoglobulin (IVIG) therapy to improve neuromuscular disability and impairment in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). Gammagard Liquid can be used as induction therapy, which includes an induction dose followed by maintenance doses.

CIDP is a rare neuromuscular disorder affecting the peripheral nervous system. It is characterized by progressive symptoms such as weakness, tingling or loss of feeling in the limbs, loss of reflexes and difficulty walking. Infusion with immune globulin provides antibodies to block the immune and inflammatory process that destroys myelin.

Mamatha Pasnoor, M.D.

“As the standard of care for the treatment of CIDP, IG therapy is thought to help normalize compromised immune systems through immunomodulatory mechanisms,” Mamatha Pasnoor, M.D., professor in the Department of Neurology at the University of Kansas Medical Center, said in a press release. “Because CIDP is a progressive and complex disease, multiple treatment options are needed, and clinicians now have an additional therapy that can help adults with CIDP manage their disease.”

The approval is based on results from a prospective clinical study (ADVANCE-CIDP 2) that evaluated the efficacy and safety of Gammagard Liquid. Enrolled patients included adults with chronic inflammatory demyelinating polyneuropathy who relapsed in the study evaluating HqQvia (ADVANCE-CIDP 1). FDA recently approved Takeda’s HyQvia, an immune globulin infusion with recombinant human hyaluronidase. The hyaluronidase part facilitates the dispersion and absorption of immune globulin in the subcutaneous space between the skin and the muscle.

Related: FDA Approves HyQvia as Maintenance Therapy for Rare Neuromuscular Disorder

Efficacy in ADVANCE-CIDP 2 studying Gammagard Liquid was based on responder rate, which was 94.4% of the 18 patients enrolled. Improvement in grip strength and change in Rasch-built Overall Disability Scale score was seen across all patients. The most common adverse reactions observed were headache, pyrexia, anemia, leukopenia, neutropenia, illness, blood creatinine increased, dizziness, migraine, somnolence, tremor, nasal dryness, abdominal pain upper, vomiting, chills, nasopharyngitis and pain in extremity.

Gammagard Liquid is also approved to improve neuromuscular disability and impairment in adult patients with CIDP, as a replacement therapy for primary immunodeficiency (PI) in adult and pediatric patients two years of age and older, and as a maintenance therapy to improve muscle strength and disability in adult patients with multifocal motor neuropathy.

Prices for Gammagard injection range from $161.45 for one vial of 1 gm/10 mL to $4,843.50 for one vial of 30 gm/300 mL. A Takeda spokesperson said the company prices its medicines in line with value-based pricing principles. “Takeda pricing principles consider variability in affordability, healthcare system maturity, out of pocket expenditure, GDP as well as other healthcare indicators across geographies,” she said. Qualifying patients with commercial insurance may be eligible for enrollment in the Takeda Patient Support Co-Pay Assistance Program.