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Sep 19, 2018

New Drug Offers Hope for Patients with a Rare Form of Cardiomyopathy

Breakthrough therapy appears both safe and effective in patients with a rare and fatal heart condition called transthyretin amyloid cardiomyopathy.

A new drug, tafamidis, offers hope for patients with a rare form of cardiomyopathy, based on results of an international study that tested a breakthrough therapy in patients with transthyretin amyloid cardiomyopathy.

Transthyretin amyloid cardiomyopathy is a rare and fatal condition associated with progressive heart failure and causes problems with the heart’s muscle. It typically presents between the ages of 50 and 80 years and leads to debilitating symptoms like shortness of breath, fatigue and dizziness.

With no current medications approved for the treatment of this condition, average survival time is three to five years after diagnosis. However, findings recently published in the New England Journal of Medicine offer hope for patients living with this rare condition.

Known as the ATTR-ACT trial, this study tested tafamidis in 441 older patients diagnosed with transthyretin amyloid cardiomyopathy. Through the trial, participants were randomly assigned to take either 20 mg or 80 mg of tafamidis daily or a placebo pill that contained no active ingredients. The study lasted two and a half years, during which time researchers tracked key outcomes like hospitalizations, quality of life and death.

The trial was conducted across 48 medical centers in 13 countries and results were presented at the ESC Congress 2018.

Overall, researchers found that participants taking tafamidis were 32% less likely to be hospitalized and had 30% lower risk of death after 2.5 years than those taking the placebo. Tafamidis was also associated with a significantly better quality of life, which was measured by a six-minute walk test and a symptoms questionnaire.

Authors note that rates of adverse events were similar among both groups, suggesting that the drug is safe with no risk of increased complications.

“There are no medications specifically approved for the treatment of transthyretin amyloid cardiomyopathy,” said lead study investigator, Professor Claudio Rapezzi, University of Bologna, Italy. “Tafamidis improved survival and quality of life, and reduced hospitalizations, indicating that it could be an effective therapy for these patients.”

Of course, additional research is still needed to better understand how tafamidis works and when patients should start taking it after diagnosis.  However, authors note that the maker of the drug is seeking marketing approval based on study findings. And if approved, tafamidis would be the first drug specifically offered to treat transthyretin amyloid cardiomyopathy.


Questions for You to Consider

  • What is transthyretin amyloid cardiomyopathy?

  • Transthyretin amyloid cardiomyopathy is a rare and fatal heart condition, which causes symptoms similar to heart failure. It can be hereditary or acquired and is typically diagnosed in adults between 50 and 80 years old. While treatment typically includes therapies to minimize symptoms like shortness of breath and fatigue, new drugs are currently being tested to improve survival and outcomes for patients.

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