Individual results may vary
Amalia, an Alnylam Patient Ambassador on GIVLAARI
Individual results may vary
Amalia, an Alnylam Patient Ambassador on GIVLAARI
GIVLAARI® (givosiran) study results
In a 6-month study, GIVLAARI reduced AHP attacks and days of hemin use compared with placebo
In the study, 48 patients received GIVLAARI and 46 patients received placebo (an injection that did not contain any medicine) once monthly over a 6-month period. During the study, patients were able to use hemin if they experienced an attack. Using hemin to prevent an attack was not allowed during the study.
Patients taking GIVLAARI experienced fewer AHP attacks on average compared with those taking a placebo
Patients on GIVLAARI had an average of 1.9 AHP attacks compared with 6.5 in patients on placebo
Attacks were defined as those that required hospitalization, urgent healthcare visit, or intravenous (IV) hemin at home.
Patients taking GIVLAARI needed less hemin on average to treat AHP attacks compared with those taking a placebo
Patients on GIVLAARI had an average of 4.7 days of hemin use compared with 12.8 in patients on placebo
Fewer patients experienced AHP attacks over 36 months
After the 6-month study, all 93 eligible patients who remained in the study received GIVLAARI once a month. The graph below shows the 48 patients who were treated with GIVLAARI in the 6-month study and continued on in the study. Over time, fewer patients had attacks.
Attacks measured in the study were defined as those that required hospitalization, urgent healthcare visits, or IV hemin at home.
These results were observed in the clinical trial. Keep in mind that everyone responds to GIVLAARI differently.
GIVLAARI safety profile
Safety during the first 6 months of the study
- In the first 6 months of the study, 1 patient taking GIVLAARI stopped treatment due to changes in liver function. No patients taking placebo stopped treatment
The most common side effects in patients treated with GIVLAARI compared with those taking placebo in the first 6 months of the study were:
| GIVLAARI (48 patients) |
Placebo (46 patients) |
|
| Nausea | 27% | 11% |
| Injection site reactions | 25% | 0% |
| Rash | 17% | 4% |
| Changes in kidney function | 15% | 4% |
| Changes in liver function | 13% | 2% |
| Fatigue | 10% | 4% |
Safety after the first 6 months
- The most common treatment-related side effects (in ≥10% of patients) were injection site reactions (32% [30/94] patients), nausea (21% [20/94]), and fatigue (14% [13/94])
- Serious side effects were reported in 7 patients (7% of patients). Serious side effects that were reported in 2 or more patients were increased blood homocysteine (2 patients) and serious side effects related to elevated liver function tests (increased liver enzymes and an abnormal liver function test in 1 patient each)
- During this period, there was 1 death from an aortic dissection unrelated to GIVLAARI treatment
- After the initial 6-month study, 3 patients stopped treatment due to side effects. One stopped because of a severe allergic reaction, and the other 2 stopped due to increased blood homocysteine
- During this period, increased blood homocysteine levels were found in 15 of 93 patients (16%)
