Important clinical efficacy outcomes and side effects of givosiran therapy
| . | Six-month data from ENVISION study∗ . |
|---|---|
| Important clinical efficacy outcomes of givosiran treatment | |
| Reduced annualized composite porphyria attack rate | 74% reduction compared with placebo 3.2 (95% CI, 2.3-4.6) vs 12.5 (95% CI, 9.4-16.8) in patients treated with placebo |
| Decreased urinary ALA and PBG levels | 86% and 91% median percent decrease for urinary ALA and PBG, respectively |
| Decreased annualized number of days of hemin usage | Mean (95% CI), 6.8 (4.2-10.9) vs 29.7 (18.4-47.9) in patients treated with placebo, P < .001 |
| Improved patient-reported outcomes | Refer to the text for details |
| Decrease in daily worst score for pain | |
| Decrease in number of patients on and proportion of days with opioid use | |
| Improvement in physical, mental health, and QoL scores† | |
| Potential side effects of givosiran | |
| Nausea | 27% vs 11% of patients treated with placebo |
| Injection-site reactions | 25% vs 0% of patients treated with placebo |
| Increased serum creatinine or decreased eGFR | 15% vs 4% of patients treated with placebo |
| ALT >3× the upper limit of normal | 15% vs 2% of patients treated with placebo |
| Elevated homocysteine levels | Not reported on in this study |
| Rare drug-drug interactions | Not reported on in this study |
| . | Six-month data from ENVISION study∗ . |
|---|---|
| Important clinical efficacy outcomes of givosiran treatment | |
| Reduced annualized composite porphyria attack rate | 74% reduction compared with placebo 3.2 (95% CI, 2.3-4.6) vs 12.5 (95% CI, 9.4-16.8) in patients treated with placebo |
| Decreased urinary ALA and PBG levels | 86% and 91% median percent decrease for urinary ALA and PBG, respectively |
| Decreased annualized number of days of hemin usage | Mean (95% CI), 6.8 (4.2-10.9) vs 29.7 (18.4-47.9) in patients treated with placebo, P < .001 |
| Improved patient-reported outcomes | Refer to the text for details |
| Decrease in daily worst score for pain | |
| Decrease in number of patients on and proportion of days with opioid use | |
| Improvement in physical, mental health, and QoL scores† | |
| Potential side effects of givosiran | |
| Nausea | 27% vs 11% of patients treated with placebo |
| Injection-site reactions | 25% vs 0% of patients treated with placebo |
| Increased serum creatinine or decreased eGFR | 15% vs 4% of patients treated with placebo |
| ALT >3× the upper limit of normal | 15% vs 2% of patients treated with placebo |
| Elevated homocysteine levels | Not reported on in this study |
| Rare drug-drug interactions | Not reported on in this study |
A composite porphyria attack was defined as an attack that resulted in hospitalization, an urgent health care visit, or intravenous administration of hemin at home.
CI, confidence interval; eGFR, estimated glomerular filtration rate; QoL, quality of life.
Derived from Balwani et al.63
Assessed by SF-12 Physical Component Summary, Mental Component Summary, EuroQol 5D.