Risk of mortality in a rare cardiac condition, transthyretin amyloid cardiomyopathy, plunged by 30% on administration of Pfizer’s tafamidis in a 30-month long study on 440 patients.

As per the report presented at the European Society of Cardiology Congress in Munich by Pfizer, all-cause mortality spanning over 30 months was decreased by tafamidis in transthyretin amyloid cardiomyopathy patients by 29.5% against 42.9% in patients on placebo. The rate of admission into hospitals due to cardiovascular causes also fell by 32%.

 “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 diseases at Pfizer. “It’s a uniformly fatal disease.”

This rare and fatal disease has no approved treatment to date. Worldwide, about 2000 to 5000 patients are presently diagnosed with transthyretin cardiomyopathy. However, 400,000 to 500,000 patients could actually be living with the condition in developed countries throughout the world and the condition remains largely underdiagnosed, opines Pfizer.

Cooperstone noted that merely 0.5 percent to 1 percent of the cases are diagnosed presently. “Part of that is driven by the fact that today there is no therapy - there’s no reason to actually make the diagnosis,” she commented, suggesting that a potential medicine could increase the rate of diagnosis.

Once Pfizer revealed the findings from the research, the drug was granted the breakthrough designation in May by the U.S. Food and Drug Administration. Till it bags the approval, Pfizer has secured a way to release the drug for use by patients before the regulatory sanction.

John Boris, SunTrust Robinson Humphrey analyst commented in a recent research paper that if the price is labeled at $150,000 to $300,000 per year, $600 million to $1.2 billion revenue could be generated by the drug from international markets. He also mentioned that the drug could be launched after gaining approval by 2019.