Pfizer announced that their investigational treatment tafamidis, for the rare disease transthyretin cardiomyopathy (TTR-CM), has been granted Fast Track designation by the Food and Drug Administration (FDA).

Tafamidis (trade name ‘Vyndaqel’), a novel TTR stabilizer, is currently being evaluated by the company in the Phase 3 clinical trial, ATTR-ACT, expected to be completed in the first half of 2018. The trial includes patients with variant transthyretin familial amyloid cardiomyopathy (TTR-FAC), the hereditary form of the disease, and those with wild-type TTR-CM, which is not hereditary.

“The hereditary form of this disease not only impacts the lives of people with the disease, but also may affect multiple generations; patients often have been caregivers for a parent, and concerned for their children who also may inherit the disease,” said Isabelle Lousada, CEO and President, Amyloidosis Research Consortium. Heart disease occurs in TTR-CM patients when unstable tetramers dissociate resulting in misfolded proteins that aggregate into amyloid fibrils and deposit in the heart. 

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There are currently 1,000 diagnosed with TTR-CM in the world. The average life expectancy following diagnosis is 3 to 5 years. “We look forward to working closely with the FDA to evaluate this medicine as a potential new treatment option for patients,” said Brenda Cooperstone, SVP and CDO of Rare Disease at Pfizer.

Vyndaqel is approved in Europe and a number of other countries to treat transthyretin familial amyloid polyneuropathy (TTR-FAP) but not in the U.S.

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