Acute Intermittent Porphyria
2
Pipeline Programs
1
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
0
1
1
0
0
0
0
Early DiscoveryClinical DevelopmentMarket
Drug Modality Breakdown
RNA Therapeutic
2100%
Competitive Landscape
1 companies ranked by most advanced pipeline stage
Alnylam PharmaceuticalsCAMBRIDGE, MA
2 programs1
1
GivosiranPhase 1/2RNA Therapeutic1 trial
givosiranPhase 1RNA Therapeutic1 trial
Active Trials
Trial Timeline
Clinical trial activity over time
2021
2022
2023
2024
2025
2026
Alnylam PharmaceuticalsGivosiran
Alnylam Pharmaceuticalsgivosiran
Clinical Trials (2)
Total enrollment: 56 patients across 2 trials
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
Start: Oct 2016Est. completion: Nov 202116 patients
Phase 1/2Completed
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
Start: May 2015Est. completion: Sep 201740 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
1 companies competing in this space