Acute Hepatic Porphyria

Gastroenterology
1
Pipeline Programs
1
Companies
4
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
0
0
0
1
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
2100%
+ 2 programs with unclassified modality

Competitive Landscape

1 companies ranked by most advanced pipeline stage

Alnylam Pharmaceuticals
4 programs
1
GivosiranPhase 3RNA Therapeutic1 trial
ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)N/A1 trial
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)N/A1 trial
GivosiranN/ARNA Therapeutic1 trial
Active Trials
NCT04883905Recruiting150Est. Apr 2027
NCT02240784Completed136Est. Apr 2021
NCT04056481Approved For Marketing
+1 more trials

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
Alnylam PharmaceuticalsGivosiran
Alnylam PharmaceuticalsELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)
Alnylam PharmaceuticalsEXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

Clinical Trials (4)

Total enrollment: 380 patients across 4 trials

NCT03338816Alnylam PharmaceuticalsGivosiran

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

Start: Nov 2017Est. completion: May 202194 patients
Phase 3Completed
NCT04056481Alnylam PharmaceuticalsGivosiran

Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria

N/AApproved For Marketing
NCT04883905Alnylam PharmaceuticalsELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

ELEVATE, a Registry of Patients With Acute Hepatic Porphyria (AHP)

Start: Apr 2021Est. completion: Apr 2027150 patients
N/ARecruiting
NCT02240784Alnylam PharmaceuticalsEXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)

Start: Aug 2014Est. completion: Apr 2021136 patients
N/ACompleted

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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 late-stage (Phase 3) programs — potential near-term approvals
1 actively recruiting trials targeting 380 patients
1 companies competing in this space

Related Indications

Hepatic Impairment(79)Gastroesophageal Reflux Disease(37)Irritable Bowel Syndrome(29)Gastroesophageal Reflux(27)Liver Transplantation(24)Celiac Disease(23)Hepatic Insufficiency(22)Liver Cirrhosis(22)Gastroesophageal Reflux Disease (GERD)(14)Hepatic Encephalopathy(12)