Acute Intermittent Porphyria
2
Pipeline Programs
2
Companies
2
Clinical Trials
0
Approved Products
Pipeline by Development Stage
Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
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Early DiscoveryClinical DevelopmentMarket
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Chiesirecombinant human porphobilinogen deaminase
uniQurerAAV2/5-PBGD vector dosage 1
Clinical Trials (2)
Total enrollment: 44 patients across 2 trials
Porphozym in the Treatment of Acute Attacks in AIP
Start: Jun 2003Est. completion: Jun 200636 patients
Phase 2/3Completed
Phase I Gene Therapy Clinical Trial Using the Vector rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria
Start: Nov 2012Est. completion: Nov 20148 patients
Phase 1Completed
Phase Legend
Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available
Key Insights
2 companies competing in this space
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