Givlaari (givosiran) 189 mg vial for injection

Description

💊 Prescription ALAS1-directed small interfering RNA for the treatment of adults with acute hepatic porphyria.

▪︎ Givosiran 189 mg/mL, one single-dose vial; 2.5 mg/kg subcutaneously once monthly, administered by a healthcare professional

▪︎ Manufacturer: Alnylam Pharmaceuticals

▪︎ FDA approval: November 2019

🔬 How it works: haem is assembled in a chain of eight steps, and in this disease one step works poorly. Finished haem normally restrains the first enzyme of the chain, ALAS1, by feedback; when haem falls short that restraint loosens, the chain accelerates and runs into the fault, so unfinished intermediates pile up in front of it. Two of them — aminolevulinic acid and porphobilinogen — are toxic to the nervous system and are what the attacks are made of. Givosiran silences ALAS1 in liver cells, holding the first enzyme quietened continuously so the intermediates never accumulate. Intravenous hemin breaks an attack that has begun; this drug prevents it from starting. 📊 Evidence: in ENVISION (NEJM 2020) in 94 patients the annualised attack rate was 3.2 versus 12.5 on placebo — 74% lower (p<0.001) — with lower urinary intermediates, fewer days of hemin use and better daily pain scores. At the 36-month final analysis (J Hepatol 2023), 86% of patients on continuous treatment and 92% of those crossed over from placebo had no attacks at all, and annualised days of hemin use fell from 16.2 to 0.4.

⚠️ Prescription-only, given by a healthcare professional with support for anaphylaxis at hand; severe hypersensitivity to givosiran is the only contraindication. There is no boxed warning, but six precautions apply: anaphylaxis, hepatic toxicity (liver tests at baseline and periodically; dose reduced to 1.25 mg/kg after significant elevations), changes in renal function, injection-site and recall reactions, raised homocysteine (measure at baseline, consider vitamin B6) and pancreatitis — the last being easy to mistake for another porphyria attack. It does not replace hemin for an attack in progress, nor the avoidance of triggering drugs, fasting and alcohol.

📦 Ordered on request via chat — availability and current price are confirmed by the administrator.