Transthyretin Cardiomyopathy is a rare heart disease that can lead to heart failure. This weekend Pfizer presented data that shows its Phase III tafamidis therapy reduced the risk of death in patients with a distinct form of the disease by 30 percent, giving the drug the chance to become the first approved medication for these patients.
The late-stage trial tested tafamidis over a 30-month period in patients with wild-type or variant (hereditary) transthyretin amyloid cardiomyopathy (ATTR-CM). Pfizer In addition to the reduced risk of death, Pfizer said the use of tafamidis also reduced the rate of cardiovascular-related hospitalizations by 32 percent for this patient population, compared to placebo. Pfizer presented its findings this weekend during Hot Line Session 3 at the European Society of Cardiology Congress 2018 in Munich, Germany. Pfizer also published the results of the Phase III trial in the New England Journal of Medicine.
Brenda Cooperstone, chief rare disease development officer at Pfizer, said the findings from the ATTR-ACT study brings the company closer to its goal of “providing an urgently needed therapy for a serious and often fatal disease.” The patient population for this particular disease is small, about 2,500 people globally. The average life expectancy for people with transthyretin cardiomyopathy is three to five years from diagnosis.
“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,” Cooperstone said in a statement.
In March, Pfizer presented topline results from the study that shook investors in rival companies attempting to develop TTR treatments. Pfizer’s 441-patient study included those who had the hereditary form of the disease, as well as the “wild-type form,” meaning it can occur as people age. Following the announcement of topline results in May, the U.S. Food and Drug Administration (FDA) granted the drug Breakthrough Therapy Designation. Last year the FDA granted Fast Track designation to Tafamidis for transthyretin cardiomyopathy.
Cooperstone told Reuters it anticipates submitting its application to the FDA in the fourth quarter of this year.
Pfizer has tried for approval for of Tafamidis before as a treatment for a neurodegenerative disease, but was soundly rejected by the U.S. Food and Drug Administration. Pfizer acquired Tafamidis in 2010 as part of the deal for FoldRx. If approved, Pfizer said it has established an expanded access treatment protocol to make tafamidis available to ATTR-CM patients. Tafamidis has been approved in Europe for transthyretin familial amyloid polyneuropathy (TTR-FAP). It is sold there under the brand name Vyndaqel.
For this indication, tafamidis would not have any competing drugs, but there are drugs for a similar disease on the market and in development. Earlier this month the FDA approved rival Alnylam’s Onpattro (patisiran) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. It is the first and only RNA interference (RNAi) therapeutic to ever be approved. hATTR amyloidosis is a rare disease that affects about 50,000 people worldwide.
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