Givlaari lowers rate of porphyria attacks
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Patients with acute hepatic porphyria treated with Givlaari experienced a lower rate of porphyria attacks, according to results of the ENVISION study.
Givlaari (givosiran, Alnylam) is a GaINAc-conjugate RNAi therapeutic that targets aminolevulinic acid synase 1 (ALAS1) for the treatment of acute hepatic porphyrias (AHP).
“Patients with AHP suffer through debilitating and potentially life-threatening attacks, and for some, chronic pain between attacks, resulting in a diminished quality of life and ability to function day-to-day,” Manisha Balwani, MD, MS, of the Icahn School of Medicine at Mount Sinai, said in a press release.
In the phase 3 ENVISION trial, researchers randomly assigned symptomatic patients with AHP to receive either subcutaneous givosiran (2.5 mg/kg; n = 48) or placebo (n = 46) monthly for 6 months. The primary endpoint of the study was the annualized rate of composite porphyria attacks (attacks that resulted in hospitalization, urgent health care visit or intravenous administration of hemin at home) with acute intermittent porphyria, a common subtype of AHP.
Among 89 patients with acute intermittent porphyria, the mean annualized attack rate was 3.2 in patients who received givosiran compared with 12.5 in the placebo group (P < .001). Results were similar among the 94 total patients with AHP.
Patients who received givosiran also experienced lower levels of urinary aminolevulinic acid and porphobilinogen, fewer days of hemin use and better daily pain scores compared with patients who received placebo.
Investigators found that the efficacy of givosiran came with a higher frequency of hepatic and renal adverse events, including elevated serum aminotransferase levels, changes in serum creatinine levels, estimated glomerular filtration rate and injection-site reactions.
“We believe the publication of the ENVISION phase 3 study results in [The New England Journal of Medicine] further underscores the clinical benefit of givosiran,” Balwani said in the release. “The significant reduction in annualized attack rate, paired with improvement in multiple other disease manifestations in patients experiencing ongoing attacks, demonstrate the potential therapeutic impact of givosiran for patients afflicted with AHP.”
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