Phase III data from post-hoc study of Vyndaqel in transthyretin amyloid cardiomyopathy published in Circulation: Heart Failure.

This analysis from the Phase III Transthyretin Amyloid Cardiomyopathy Clinical Trial (ATTR-ACT) and its long-term extension (LTE) study was published in Circulation: Heart Failure. In ATTR-ACT, patients were randomized to receive VYNDAQEL 80 mg, 20 mg or placebo, and upon study completion at 30 months could enroll in the LTE study. In the LTE study, patients who had been treated with Vyndaqel 80 mg continued this therapy, then transitioned to the bioequivalent single-capsule Vyndamax. Patients treated with placebo in ATTR-ACT were randomized to receive either Vyndaqel 80 mg or 20 mg in the LTE study and were subsequently transitioned to Vyndamax. Vyndamax 61 mg is bioequivalent to Vyndaqel 80 mg but is not interchangeable on a per-mg basis.

In ATTR-ACT, treatment with Vyndaqel demonstrated a 30% reduction in mortality at 30 months compared to placebo. With a median follow up of nearly five years, the analysis published in Circulation: Heart Failure showed a clinically significant 41% reduction in the risk of all-cause mortality among patients who received continuous Vyndaqel /Vyndamax treatment (median follow up 58.5 months) compared to patients who first received placebo in ATTR-ACT before transitioning to Vyndaqel /Vyndamax in the LTE (median follow up 57.1 months; HR: 0.59; 95% CI: 0.44–0.79; P<0.001). median survival was 67 months (95% ci: 47.0–n e) in the continuous treatment arm compared to 35.8 months (95% ci: 29.7–41.1) in the placebo to treatment arm. the preliminary five-year survival rate was 53.2% in the continuous treatment arm versus 32.4% in the placebo to treatment arm.

See: "Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy" Perry Elliott et al. Circulation: Heart Failure, 20 Dec 2021 https://doi.org/10.1161/CIRCHEARTFAILURE.120.008193