Significantly reduce death, large-scale research in the past 3 years confirmed the efficacy of new Pfizer drugs

April 02, 2018 Source: WuXi PharmaTech

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Recently, Pfizer announced that its innovative therapy, tafamidis, reached its primary endpoint in a phase III clinical trial of transthyretin (TTR) amyloidosis cardiomyopathy (transthyretin cardiomyopathy). This nearly three-year trial showed that tafamidis significantly reduced the risk of death in patients.

In the human body, transthyretin has an important physiological role. Under normal conditions, these proteins consist of four identical subunits. However, when such proteins develop lesions and fold errors occur, the tetramer becomes unstable, causing misfolded proteins to accumulate in the amyloid deposit, which accumulates in the heart, with serious consequences. It is estimated that patients with confirmed transthyretin amyloidosis have an average lifespan of only 3-5 years. The tafamidis brought by Pfizer is expected to bring the gospel to these patients. This new drug is a new class of transthyretin stabilizers that are expected to improve the condition of patients.

The potential of this new drug has been validated in a phase 3 clinical trial. The researchers enrolled 441 patients, one taking 20 mg of tafamidis daily, the second group taking 80 mg of tafamidis daily, and the third group receiving placebo. The primary objective of the study was to compare patients taking tafamidis with placebo for up to 30 months and for differences in all-cause mortality and cardiovascular-related hospitalization. The results show that tafamidis significantly reduces these two indicators.

â–²The molecular structure of Tafamidis (Source: By Anypodetos (Own work) [CC0], via Wikimedia Commons)

“These top-line results are very important for patients, and we are one step closer to the goal of bringing this innovative therapy to the patient,” said Brenda Cooperstone, Pfizer Senior Vice President and Chief Development Officer, Rare Diseases, Global Product Development The PhD said: "Pfizer's rare disease department has been at the forefront to deepen understanding of transthyretin amyloidosis. We are grateful to the patients and their families involved in these clinical trials, and to the doctors and research centers in clinical trials. Contribution. We look forward to sharing more detailed results with the cardiovascular community and discussing these data with regulators to find the right regulatory path."

“Our findings have brought real hope to these patients and their families,” said Professor Mat Maurer of Columbia University. “As medical experts, all we can do now is to control the symptoms of the disease without effective drug treatment. The drugs for treating this disease are in short supply."

In 2011, tafamidis was granted orphan drug status by the US FDA. In 2017, the US FDA granted this new drug fast track qualification. The release of the results of the recent three issues has made this new drug a step closer to the market. We expect patients to come up with innovative therapies and save their lives.

Reference materials:

[1] Pfizer Announces Positive Topline Results From Phase 3 ATTR-ACT Study Of Tafamidis In Patients With Transthyretin Cardiomyopathy

[2] Pfizer Receives FDA Fast Track Designation for Tafamidis for Transthyretin Cardiomyopathy

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