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Gamunex-C

Phase 3

Primary Immunodeficiency | Monoclonal antibody | Other |Grifols, S.A.|Last Updated: Jun 5, 2023

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
ACTIVE_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment33
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04561115A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Immune Globulin (Human) 10% (Gamunex-C) PEG Process (IVIG-PEG) Compared to Gamunex-C in Participants With Primary Humoral ImmunodeficiencyPHASE3 COMPLETED 33Sep 2, 2020Mar 28, 2022Jun 5, 202310 United States
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Study Endpoints
Primary Endpoints
AUC (0-7): Area Under the Concentration Time Curve Over a Dosing Interval of Either Every 3 Weeks [AUC0-21 Days] or Every 4 Weeks [AUC0-28 Days] for Total Immunoglobulin G (IgG)
Pre-dose, within 10 minutes of last infusion completion, at 1, 3, 6, 24, 48 hours and 4, 7, 14 and 21 days (up to 3 weeks), and 28 days (up to 4 weeks - only for subjects on a 4-week dosing schedule) post-infusion

AUC (0-7 days) is calculated as AUC (0-21 days)/3 for subjects with a dosing frequency of every 3 weeks and as AUC(0-28 days)/4 for subjects with a dosing frequency of every 4 weeks.

Cmax: Maximum Concentration in a Dosing Interval for Total IgG
Pre-dose, within 10 minutes of last infusion completion, at 1, 3, 6, 24, 48 hours, and 4, 7, 14, 21 and 28 days post-infusion (up to 4 weeks)
Secondary Endpoints
Rate of Serious Bacterial Infections (SBIs)
Baseline up to 6 months for each period, i.e., overall GC treatment phase and overall IVIG-PEG treatment phase with an aggregate duration of up to 12 months
Rate of Events Per Participant Per Year in Participants With Any Kind of Infection
Baseline up to 6 months for each period, i.e., overall GC treatment phase and overall IVIG-PEG treatment phase with an aggregate duration of up to 12 months
Rate of Days Per Person Per Year That Participants Were on Antibiotics
Baseline up to 6 months for each period, i.e., overall GC treatment phase and overall IVIG-PEG treatment phase with an aggregate duration of up to 12 months
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Gamunex-CACTIVE_COMPARATORParticipants received Gamunex-C by means of an infusion pump at a dose of 200 to 800 mg/kg per infusion at an infusion rate of 1 mg/kg/min or up to 8 mg/kg/min depending on participant tolerance. The participant's usual mg/kg dose (given on either a 3 or 4 week repeating schedule) was the same mg/kg dose and schedule that the participant was receiving prior to entering screening. This mg/kg dose and schedule were used throughout the study duration. Gamunex-C was administered every 3 weeks (±4 days) or 4 weeks (±4 days), depending on the participant's prior IVIG dosing schedule. The duration of Gamunex-C treatment included the Gamunex-C PK Phase (up to 4 weeks) plus the additional Gamunex-C Run-in Phase (up to 4.5 months) for participants not receiving Gamunex-C or not on a stable dose of Gamunex-C upon entering the trial. The approximate maximum duration was up to 6 months.
IVIG-PEGEXPERIMENTALParticipants received IVIG-PEG by means of an infusion pump at a dose of 200 to 800 mg/kg per infusion at an infusion rate of 1 mg/kg/min or up to 8 mg/kg/min depending on participant tolerance. IVIG-PEG was administered every 3 weeks (±4 days) or 4 weeks (±4 days), depending on the participant's prior IVIG dosing schedule. The duration of IVIG-PEG treatment included the IVIG-PEG Treatment Phase (up to 4.5 months) and the IVIG-PEG PK Phase (up to 4 weeks), for an approximate maximum of up to 6 months.
Interventions
NameTypeDescription
Gamunex-CBIOLOGICALIntravenous infusion.
IVIG-PEGBIOLOGICALIntravenous infusion.
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites10

Inclusion Criteria: * Male or female between 18 and 75 years of age (inclusive) at Screening * Documented and confirmed pre-existing diagnosis of PI with features of hypogammaglobulinemia requiring IV IgG replacement therapy including but not limited to the following humoral-based immunodeficiency ...

Countries:United States
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