What Is Givlaari? Uses, Dosage, and What You Need to Know in 2026

Givlaari (givosiran) is a prescription medication used to treat adults with acute hepatic porphyria (AHP), a group of rare, life-threatening genetic diseases that can cause severe attacks involving abdominal pain, neurological symptoms, and even respiratory failure. Approved by the FDA on November 20, 2019, Givlaari was a landmark moment in medicine — it was the first disease-modifying therapy to target the root cause of AHP rather than just managing symptoms after attacks occur.

What Condition Does Givlaari Treat?

Givlaari is FDA-approved for the treatment of acute hepatic porphyria (AHP) in adults. AHP includes four types:

• Acute Intermittent Porphyria (AIP) — The most common form, accounting for approximately 80% of all AHP cases
• Variegate Porphyria (VP) — Causes both neurovisceral attacks and skin photosensitivity
• Hereditary Coproporphyria (HCP) — Similar to AIP but caused by a deficiency in a different enzyme
• ALA Dehydratase Deficient Porphyria (ADP) — The rarest form of AHP

In all of these conditions, episodes called porphyria attacks can occur suddenly — causing severe abdominal pain, nausea and vomiting, constipation, muscle weakness, pain in the limbs or back, seizures, mental status changes, and in severe cases, respiratory failure. These attacks can require hospitalization, IV medication, and prolonged recovery. They can also cause permanent neurological damage.

Who Takes Givlaari?

Givlaari is approved for adults (18 years and older) with AHP who have active disease — meaning they are experiencing porphyria attacks that require medical intervention. Most payers require documentation of at least 2 attacks in the prior 6 months requiring hospitalization, urgent care, or IV hemin before approving coverage.

AHP is more common in women, particularly during their childbearing years when hormonal changes can trigger attacks. The disease often begins to manifest in the third or fourth decade of life. It affects an estimated 1 in 50,000 people worldwide, though many cases are undiagnosed or misdiagnosed for years.

What Form Does Givlaari Come In?

Givlaari is supplied as a sterile, preservative-free injectable solution at 189 mg/mL in a single-dose 1-mL vial. The solution is colorless to yellow. It is given as a subcutaneous injection (under the skin) — typically into the abdomen, the back or side of the upper arms, or the thighs. The injection site is rotated each month. An injection should never be given into scar tissue, reddened, inflamed, or swollen areas.

Givlaari Dosage: How Much and How Often?

The recommended dose of Givlaari is 2.5 mg/kg of body weight, administered once monthly via subcutaneous injection. Dosing is based on your actual body weight, which means if your weight changes significantly, your dose may need to be recalculated.

Importantly, Givlaari must be administered by a healthcare professional. You cannot self-inject at home (unlike some other subcutaneous biologics). Your monthly injection will be given at an approved administration site — which may be your specialist's office, an infusion center, or via a home nursing visit in some areas.

If liver enzyme levels (ALT) become significantly elevated, the dose may be reduced to 1.25 mg/kg once monthly until levels normalize, at which point the full dose of 2.5 mg/kg may be resumed.

Is Givlaari a Controlled Substance?

No. Givlaari (givosiran) is not a controlled substance. It does not appear on the DEA's schedule of controlled substances. There are no restrictions on refills based on controlled substance status, though prior authorization is required by insurers.

How Well Does Givlaari Work?

In the pivotal ENVISION clinical trial, patients with AHP who received Givlaari experienced approximately 70% fewer porphyria attacks compared to those receiving placebo. In the subset of patients with AIP (the most common subtype), the reduction in annualized attack rate was 74%. Long-term follow-up data at 36 months showed:

• 86-96% of patients had zero attacks in the final 3 months of follow-up
• Sustained reductions in ALA and PBG (the toxic compounds that cause attacks)
• Sustained improvements in quality of life

What Is Givlaari's Manufacturer?

Givlaari is manufactured and marketed by Alnylam Pharmaceuticals, Inc., based in Cambridge, Massachusetts. Alnylam is one of the pioneers of RNA interference (RNAi) therapeutics. Givlaari was only the second RNAi drug to receive FDA approval (following Alnylam's Onpattro in 2018). It is made at Ajinomoto Althea, Inc. in San Diego, California.

Want to understand exactly how Givlaari works at the molecular level? See our plain-English explanation: How Does Givlaari Work?

If you are prescribed Givlaari and need help finding a specialty pharmacy or administration site, medfinder calls sites on your behalf and texts you the results.