Regulatory Decision Summary for Cuvitru

Cuvitru is based on the currently licensed Immune Globulin Infusion (IGI, Human) 10% solution (GAMMAGARD Liquid) for the intravenous (IV) and subcutaneous (SC) replacement therapy of Primary Immunodeficiency Disease (PID). The production of IGSC 20% follows the same manufacturing processes as IGI 10% except for ultra-/diafiltration and final formulation at 20% weight/volume (w/v) protein concentration.

The clinical safety, efficacy and pharmacokinetics (PK) data presented in this dossier are from three completed clinical trials. The two main studies: study 170904 in North America and study 170903 in Europe were conducted with IGSC 20% in adult and pediatric patients with PID; the third study was conducted with a 10% Ig G solution administered SC (IGSC 10%) in adult and pediatric patients with PID.

Cuvitru is efficacious as demonstrated by the high trough levels maintained in general during studies, low validated acute serious bacterial infections rates. The maximum recommended infusion rate of 60 mL/hr/site was well tolerated leading to less duration of infusion and less number of infusion sites. Quality of life seems improved in PID patients who needed replacement therapy and appreciated treatment with more satisfaction. As showed in the analysis of the safety in the different clinical studies and in the Integrated Summary of Safety (ISS) which summarizes clinical data relevant to the safety and tolerance of IGSC, 20% in subjects with PID aged 2 years or older who participated in the cited clinical studies the safety profile of ISCG 20% is acceptable.

Overall the benefit/risk assessment for Cuvitru is favorable and a notice of compliance was recommended.