Recent Updates
Recently added Catalysts

i.v. recombinant human C1 inhibitor

Phase 2

Genetic Disorders | Small molecule | Rare Disease |Pharming Group N.V.|Last Updated: Apr 25, 2013

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
UNCONTROLLEDDMC
Total Trials2
Total Enrollment28
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00262288Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary AngioedemaPHASE2 COMPLETED 14Apr 1, 2004Jan 1, 2007Feb 22, 20131 Netherlands
NCT00261053Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary AngioedemaPHASE2 COMPLETED 14Jun 1, 2003Dec 1, 2005Apr 25, 20131 Netherlands
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Primary outcomes: Relief of angioedema symptoms
24 hours
Secondary Endpoints
Secondary outcomes: Safety and tolerability; pharmacokinetics/pharmacodynamics
90 days
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Recombinant Human C1INHEXPERIMENTAL -
1OTHEROpen-label i.v. administration of 100 U/kg rhC1INH
Interventions
NameTypeDescription
i.v. recombinant human C1 inhibitorDRUG -
Unlock Study Design Details
Eligibility Criteria
Age Range16 Years — 70 Years
SexALL
Healthy VolunteersNo
Study Sites1

Main inclusion Criteria: * Clinical and laboratory diagnosis of HAE * Plasma level of functional C1INH of less than 50% of normal * Severe attack of abdominal, facial-oro-pharyngeal, genito-urinary and/or peripheral HAE. Main exclusion Criteria: * Acquired angioedema * Pregnancy or breastfeeding ...

Countries:Netherlands
Unlock Eligibility Criteria