Rare heart disease drug reduces risk of death by 30 percent in study

Reuters , Monday 27 Aug 2018

The data was presented at the European Society of Cardiology Congress in Munich


Pfizer Inc said on Monday that its drug tafamidis reduced the risk of death for patients with a rare and fatal heart disease by around 30 percent, boosting the prospects of what could be a billion-dollar-a-year drug. 

The study showed that tafamidis reduced all-cause mortality over a 30-month period in patients with transthyretin amyloid cardiomyopathy to 29.5 percent, versus 42.9 percent in patients who received a placebo. It also reduced the rate of cardiovascular-related hospitalization by around 32 percent, Pfizer said.

The data was presented at the European Society of Cardiology Congress in Munich.

“The result is really, phenomenally encouraging and gives fantastic hope for patients with this devastating illness,” said Dr. Brenda Cooperstone, chief development officer of rare disease at Pfizer. “It’s a uniformly fatal disease.” 

Tafamidis is one of 15 potential blockbuster treatments Pfizer is trying to develop. The company’s chief executive officer, Ian Read, has touted Pfizer’s pipeline, arguing that the growth potential from these drugs makes undertaking a large transaction unnecessary. 

But Pfizer believes the disease is underdiagnosed and that there could be 400,000 to 500,000 patients in developed markets around the world. Cooperstone said the current diagnosis rate is only around 0.5 percent to 1 percent.

“Part of that is driven by the fact that today there is no therapy - there’s no reason to actually make the diagnosis,” she said, noting that a real treatment should significantly help improve the diagnosis rate. 

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