Tafamidis phase III trial positive results for treatment of transthyretin amyloid cardiomyopathy. - Pfizer

Pfizer Inc. has announced the primary results from the Tafamidis Phase III Transthyretin Cardiomyopathy (ATTR-ACT) study which showed tafamidis significantly reduced the hierarchical combination of both all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period (P=0.0006) in patients with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a rare, fatal, and underdiagnosed condition associated with progressive heart failure for which there are no approved pharmacologic treatments. The late-breaking findings were presented during Hot Line Session 3 at the ESC Congress 2018 in Munich, Germany and simultaneously published online in the New England Journal of Medicine (NEJM).

The ATTR-ACT study showed tafamidis significantly reduced all-cause mortality (29.5% vs. 42.9%; hazard ratio = 0.70, 95% confidence interval [CI] 0.51-0.96, P=0.0259) and cardiovascular-related hospitalizations (0.48 vs 0.70 annualized rate; relative risk ratio = 0.68, 95% CI 0.56-0.81, P<0.0001), compared to placebo. this represents a 30 reduction in the risk of mortality and 32 reduction in the rate of cardiovascular-related hospitalization. the findings also showed a consistent directional mortality benefit of tafamidis across all sub-groups. secondary study endpoints also showed tafamidis reduced the decline in the six minute walk test distance p><0.0001), a measure of functional capacity and reduced the decline in aspects of quality of life measured by the kansas city cardiomyopathy questionnaire overall score p><0.0001), compared with placebo at month 30. tafamidis was also well tolerated with an observed safety profile comparable to placebo.>

The NEJM manuscript, titled �Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy,� will be published in the September 13 printed issue of NEJM.

In light of the seriousness of the disease and the lack of pharmacologic treatment options, Pfizer has established an expanded access treatment protocol to make tafamidis available to ATTR-CM patients who may benefit from treatment prior to regulatory approval. The expanded access treatment protocol is posted on clinicaltrials.gov (NCT02791230), and additional information about requesting access may be found at www.pfizercares.com. Access to these programs may vary by country; physicians may contact their local Pfizer Medical department for further information. Interested ATTR-CM patients should contact their local physician to discuss whether accessing tafamidis may be an appropriate option.

See- "Tafamidis Treatment for Patients with Transthyretin Amyloid Cardiomyopathy"- Mathew S. Maurer, M.D., Jeffrey H. Schwartz, Ph.D., Balarama Gundapaneni, M.S., Perry M. Elliott, M.D. et al.,August 27, 2018 DOI: 10.1056/NEJMoa1805689.