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Efficacy and Safety Profile


Vyndaqel® delayed progression of neurological impairment and reduced decline in QOL for familial patients with stage 1 ATTR-PN4

Primary analysis: ITT

In a primary analysis of ITT patients, including patients discontinuing due to liver transplantation:5,6

>>45.3% of patients on Vyndaqel® showed no disease progression vs. 29.5% on placebo as measured by the NIS-LL endpoint.*

>>Patients on Vyndaqel® had reduced decline in QOL compared to placebo as demonstrated by TQOL score (2.0 points from baseline vs. 7.2).*

Secondary analysis: EE

In a prespecified secondary analysis of the EE population, designed to adjust for patient attrition due to liver transplantation and other discontinuations:4,6

>>Significantly more patients on Vyndaqel® showed no disease progression (60%) vs. placebo (38.1 as measured by the NIS-LL primary endpoint.)

>>Patients on Vyndaqel® had significantly reduced decline in QOL compared to placebo as demonstrated by TQOL score (0.1 points from baseline vs. 8.9).

Secondary endpoints showed delayed progression of impairment in neurological function, large-nerve fiber function, and small-nerve fiber function with Vyndaqel® vs. placebo at 18 months.4†

52%

less decline of neurological function demonstrated by

NIS-LL (p=0.03)4ⱡ

53%

less decline in large-fiber function (p=0.07)

81%

less decline in small-fiber function (p=0.01)

mBMI significantly improved for patients on Vyndaqel® (p<0.0001).4ⱡ
Significant improvement in mBMI was seen as early as 6 months (p<0.0007).4ⱡ

>>mBMI can be used as a clinical indicator to reflect disease progression and treatment response in patients with ATTR-PN.7

>>mBMI significantly worsened for patients on placebo throughout the 18-month study (p<0.0001).4

* Not statistically significant.
Analysis of ITT cohort.
In the original statistical plan, the endpoint did not adjust for multiplicity.6