Recent Updates
Recently added Catalysts

Harvoni

Phase 2

Porphyria Cutanea Tarda | Small molecule | Infectious Disease |Gilead Sciences, Inc.|Last Updated: Jul 3, 2023

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
UNCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment23
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03118674Harvoni Treatment Porphyria Cutanea TardaPHASE2 COMPLETED 23Sep 6, 2017Mar 4, 2022Jul 3, 20233 United States
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Number of Participants With Resolution of Active PCT by 7 Months After Start of Therapy
7 months

Resolution of active PCT, defined as normalization of plasma porphyrins (less than 0.9 mcg/dL) by 7 months after start of therapy

Secondary Endpoints
Time to Resolution of Active PCT
through study completion, an average of 1 year
Number of Participants With Complete Biochemical Remission of PCT
12 Months
Number of Participants With Cure of CHC
Up to 15 months
Unlock Study Endpoints
Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
HarvoniEXPERIMENTAL1 tablet per day, oral, taken with or without food. 8 weeks for patients without cirrhosis, not previously treated with HCV GT1 and HCV rNA \< 6 million IU/mL; 12 weeks for patients without cirrhosis; 24 weeks for patients with compensated cirrhosis
Interventions
NameTypeDescription
HarvoniDRUGOne capsule of Harvoni/ ledipasvir, 90 mg + sofosbuvir, 400 mg administered daily for 8, 12, or 24 weeks
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria 1. Willing and able to give informed consent 2. ≥18 years of age 3. Symptoms and signs consistent with PCT and well documented biochemical diagnosis (urinary total porphyrin excretion \> 500 mcg/g Creatinine with HPLC pattern typical of PCT-predominance of 8- and 7-carboxyl porph...

Countries:United States
Unlock Eligibility Criteria