NDASUBCUTANEOUSSOLUTIONPriority Review
Approved
Nov 2019
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
5
Data completeness
6/8 — Strong
Priority Review
GIVLAARI (givosiran sodium) by Alnylam Pharmaceuticals is double-stranded small interfering rna that causes degradation of aminolevulinate synthase 1 ( alas1 ) mrna in hepatocytes through rna interference, reducing the elevated levels of liver alas1 mrna. First approved in 2019.
Drug data last refreshed 17h ago · AI intelligence enriched 3w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
GIVLAARI (givosiran sodium) is a double-stranded small interfering RNA (siRNA) therapy approved in 2019 for acute hepatic porphyria (AHP). It works by degrading ALAS1 mRNA in hepatocytes through RNA interference, reducing neurotoxic intermediates (ALA and PBG) that drive acute attacks and disease manifestations. Administered subcutaneously, it represents a targeted genetic approach to a rare metabolic disorder.
$0.015BPart D
Peak7.9 years to LOEAt peak lifecycle with modest Part D penetration (254 claims in 2023), indicating a niche rare disease product with stable but limited commercial scale.
Mechanism of Action
double-stranded small interfering RNA that causes degradation of aminolevulinate synthase 1 ( ALAS1 ) mRNA in hepatocytes through RNA interference, reducing the elevated levels of liver ALAS1 mRNA. This leads to reduced circulating levels of neurotoxic intermediates aminolevulinic acid (ALA) and…
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (5)
A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
Started Apr 2018
10 enrolled
Acute Intermittent Porphyria (AIP)Acute Hepatic Porphyria (AHP)Porphyria, Acute Intermittent+1 more
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
Started Nov 2017
94 enrolled
Acute Hepatic PorphyriaAcute Intermittent PorphyriaPorphyria, Acute Intermittent+4 more
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
Started Oct 2016
16 enrolled
Acute Intermittent Porphyria
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
Started May 2015
40 enrolled
Acute Intermittent Porphyria
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
Acute Hepatic Porphyria
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.