Givlaari Effects Sustained Over Time for Patients with Acute Hepatic Porphyria


In 2019, Alnylam Pharmaceuticals received FDA approval for Givlaari, the first RNAi therapy for patients with acute hepatic porphyria. Although some were initially worried about the therapy’s safety and efficacy, open-label Givlaari data from the Phase 3 Envision study shows positive results. Read the full results on Alnylam’s website.

Acute Hepatic Porphyria

To understand acute hepatic porphyria, you need to first understand its nuances. Acute hepatic porphyria is part of a group of 8 rare, inherited metabolic disorders. People with porphyria don’t have enough enzymes to produce heme, a blood pigment in hemoglobin that helps transport oxygen throughout the body. As a result, porphyrins (chemicals) accumulate in the cell and reduce oxygen.

Hepatic porphyria occurs when patients lack enzymes in their liver. There are a few subtypes of acute hepatic porphyria, including acute intermittent porphyria, variagate porphyria, hereditary coproporphyria, and hereditary deficit of delta-aminolevulinic acid dehydratase.

When experiencing an “attack,” patients may present with:

  • Skin lesions, blistering, and inflammation after exposure to light
  • Muscle weakness and convulsions
  • Irritability or anxiety
  • Auditory and visual hallucinations
  • Confusion
  • Intense abdominal cramping

Attacks are often brought on from external triggers such as stress, alcohol, infections, or a poor diet. However, they can also result from hormonal changes. Learn more about hepatic porphyria here.

Givlaari Data

Initially, 6-month study data published in The New England Journal of Medicine was already positive; it presented Givlaari as an effective solution in reducing hepatic porphyria attacks when compared to a placebo. Now, the most recent study (with 1 year of data) reaffirmed those findings.

In fact, Givlaari effects, and attack reduction, were sustained over a 1-year period. Patients receiving a higher dose had better outcomes. Additionally, Givlaari seemed to be safe and well-tolerated, without many adverse reactions.

Read the source article here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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