Pfizer announced positive data from it’s phase 3 study (called ATTR-ACT), which evaluated it’s experimental therapy Tafamidis (vyndaqel) in patients with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM). Tafamidis achieved statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations.
However, the data failed to fully impress the ATTR community as some experts believed the results were not superior to other therapies. For example, Tafamidis will face competition from Alnylam Pharmaceuticals’ Onpattro (patisiran) and Ionis Pharmaceuticals’ Tegsedi (inotersen). While Onpattro received approval earlier this month, a decision on Tegsedi is expected in October in the United States. Both the drugs have been developed for treating hereditary TTR amyloidosis. Both the companies are developing their drugs for ATTR-CM.
Tafamidis, which is currently on the market in the European Union for familial amyloid polyneuropathy, was shown by the ATTR-ACT study to reduce mortality risk by around 30% and risk of hospitalizations related to cardiovascular conditions fell by 32% against placebo. The drug also boasted a safety profile similar to that of placebo.
TTR-CM is a rare disease associated with progressive heart failure and is fatal. Due to lack of treatment options, Pfizer has expanded access treatment protocol to make tafamidis accessible prior to regulatory approval to patients who may benefit from the candidate.
“We believe the ATTR-ACT study findings bring us a significant step closer to our goal of providing an urgently needed therapy for a serious and often fatal disease,” said Brenda Cooperstone, senior vice president and chief development officer for rare disease at Pfizer. “We look forward to continuing discussions with global regulatory authorities about the potential of tafamidis as a treatment option for people living with ATTR-CM.”
