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FDA Grants Breakthrough Therapy Designation for Tafamidis

FDA Grants Breakthrough Therapy Designation for Tafamidis

Pfizer recently announced that tafamidis, its investigational therapy for the treatment of patients with, transthyretin (TTR) cardiomyopathy, received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA).

This decision is supported by topline results from the tafamidis Phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study, in which tafamidis demonstrated a statistically significant reduction in the combination of all-cause mortality and frequency of cardiovascular-related hospitalizations.

As defined by the FDA, a breakthrough therapy is a drug intended to be used alone or in combination with one or more other drugs to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies. If a drug is designated as a breakthrough therapy, the FDA may expedite the development and review of such drug.

In patients with this disease, TTR breaks up and may form fibrils called amyloid. Amyloid can build up around your nerves and in other places in your body, preventing them from working normally. Eventually, the amyloid causes the symptoms of this disease.

Click here for the press release.