CONSISTENT SAFETY
The safety profile of TAKHZYRO was established in one of the largest prevention studies in HAE1-5
Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, discontinue TAKHZYRO administration and institute appropriate treatment.1
No incidence of anaphylaxis in the pivotal trial.1
Injection site reactions were the most common adverse reactions (ARs).1
every 2 weeks
(n=27)
every 4 weeks
(n=29)
(n=41)
*≥10% in any TAKHZYRO group that also occurred at a higher rate than placebo group.1
†Additional injection site reactions included hematoma, hemorrhage, pruritus, swelling, induration, paresthesia reaction, warmth, edema, and rash.1
‡Includes upper respiratory infection, viral upper respiratory infection.1
§Includes headache, tension headache, sinus headache.1
∥Includes rash, rash maculopapular, rash erythematous.1
Consistent safety profile seen in 212 patients in the open-label extension study1
Safety data of patients taking TAKHZYRO for an average of 30 months2
Hypersensitivity reactions (2%, n=4) were reported in the study.2*
Six patients discontinued due to treatment-emergent adverse events.2
Three of the subjects discontinued due to hypersensitivity reactions2
One hypersensitivity event was considered related to the study drug and led to discontinuation2
No treatment-related serious adverse events or anaphylaxis were observed.2
(N=212)
Mean study duration: 29.6 (SD=8.2) months.2
*Related, treatment-emergent hypersensitivity reactions.2
Safety profile seen in patients as young as 2 years of age1
Safety data of 21 pediatric patients taking TAKHZYRO for 52 weeks1,7
The profile of related TEAEs was similar between the 2 treatment groups.7
No deaths, serious TEAEs, hospitalizations, or discontinuations due to TEAEs were observed.7
No new safety signals were observed in these patients. Overall, the safety was similar between adult patients and pediatric patients (2 to <18 years of age).1
*TEAEs reported by ≥3 patients are presented.7
TEAE=treatment-emergent adverse event.
HR-QoL prespecified exploratory endpoint
The burden of HAE goes beyond the attack.
The unpredictable nature of HAE can affect various facets of a patient’s life.8
In the HELP study, quality of life (QoL) measures were evaluated using the AE-QoL and EQ-5D-5L questionnaires9
Limitations: These results should be interpreted with caution as they are based on patient recall and are observational/descriptive in nature. These data were also from an exploratory objective and had less evidentiary value than the primary and secondary objectives. The AE-QoL was administered to 10 adolescent patients in the study, an age group for which the instrument was not validated.9
For the EQ-5D-5L questionnaire, an instrument used to measure health status on a given day, no differences were observed. The nondisease-specific EQ-5D-5L questionnaire was administered on days 0, 98, and 182.9
Significantly more patients taking TAKHZYRO vs placebo experienced improvements in AE-QoL in the 6.5-month HELP study:
- 81% of patients receiving TAKHZYRO 300 mg Q2W (95% CI, 61-93; n=26) experienced improvement in AE-QoL total score vs 37% of patients taking placebo (P<0.05; 95% CI, 22-54; n=38)9
- Patients receiving TAKHZYRO 300 mg Q2W were 7.2 times more likely to achieve improvement in AE-QoL total score vs patients taking placebo (P<0.01)9
Definitions: The AE-QoL is a validated, angioedema-specific questionnaire in adults that was administered monthly, consisting of 4 domains (functioning, fatigue/mood, fears/shame, nutrition) and total scores. The minimal clinically important difference (MCID) is the minimum change in score that is meaningful to patients. For the AE-QoL total score, the predefined MCID is a reduction of 6 points.9-11
AE-QoL=Angioedema Quality of Life Questionnaire; EQ-5D-5L=5-level EuroQol 5-dimensional; HR-QoL=health-related quality of life; Q2W=every 2 weeks.
