Givosiran
- Generic Name
- Givosiran
- Brand Names
- Givlaari
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1639325-43-1
- Unique Ingredient Identifier
- ROV204583W
- Background
Givosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, a critical enzyme in the heme biosynthesis pathway. It is manufactured by Alnylam Pharmaceuticals and was first approved for use in the United States in November 2019 for the treatment of adults with acute hepatic porphyria, a genetic disorder in which the overprodu...
- Indication
用于治疗急性肝卟啉症（AHP）。
AHP是一类非常罕见的遗传性疾病（罕见病），其特征是使人衰弱的、潜在危及生命的发作，对一些患者来说，疾病的慢性表现会对日常功能和生活质量产生负面影响。AHP的长期并发症包括慢性神经性疼痛、高血压、慢性肾病和肝病。
- Associated Conditions
- Acute Hepatic Porphyria (AHP)
- Associated Therapies
- -
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
- Conditions
- First Posted Date
- 2019-08-14
- Last Posted Date
- 2024-05-20
- Lead Sponsor
- Alnylam Pharmaceuticals
- Registration Number
- NCT04056481
A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
Phase 1
Completed
- Conditions
- Interventions
- First Posted Date
- 2018-04-23
- Last Posted Date
- 2019-03-12
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 10
- Registration Number
- NCT03505853
- Locations
- 🇸🇪
Clinical Trial Site, Stockholm, Sweden
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
- First Posted Date
- 2017-11-09
- Last Posted Date
- 2024-04-22
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 94
- Registration Number
- NCT03338816
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
- First Posted Date
- 2016-10-31
- Last Posted Date
- 2024-03-12
- Lead Sponsor
- Alnylam Pharmaceuticals
- Target Recruit Count
- 16
- Registration Number
- NCT02949830
- Locations
- 🇬🇧
Clinical Trial Site, London, United Kingdom
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