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FDA approves first two drugs for transthyretin amyloid cardiomyopathy

Published on 07/05/19 at 09:59am

The FDA has approved its first two therapies for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM), it has emerged; the US regulator has authorised the use of Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) as a treatment to reduce cardiovascular mortality and cardiovascular-related hospitalisation associated with the condition.

The decision to approve the two drugs was based on Phase 3 data derived from the first global study into an effective treatment for the condition. The data showed that Vyndaqel “significantly reduced” the hierarchical combination of all-cause mortality and CV-related hospitalisations over 30 months compared to placebo.

Vyndaqel was also observed to have a “significant and consistent” effect on functional capacity and health status compared to placebo, with effects first noted at six months and continuing through to 30 months.  

“Pfizer's purpose is to deliver breakthrough medicines that change patients' lives. The approvals of Vyndaqel and Vyndamax deliver on this promise for patients with ATTR-CM,” said Paul Levesque, Global President, Rare Disease. “This milestone is a gamechanger for patients, who until today had no approved medicines for this rare, debilitating and fatal disease. We will continue to focus efforts on working with the physician community to increase awareness and ultimately detection and diagnosis of this disease.”

Affecting an estimated 100,000 people in the US, transthyretin amyloid cardiomyopathy is a rare and potentially fatal disease caused by the build-up of misfolded amyloid protein deposits in the heart. In the past, patients have not had access to any therapy beyond symptom management or heart transplant.

“ATTR-CM is not only fatal, but also significantly underdiagnosed, with some patients cycling through multiple doctors and a myriad of tests over a period of years while the disease progresses,” remarked Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium. “ATTR-CM is a rare disease for which more education and awareness is needed. The approval of these medicines represents an important advance for patients; however, it is equally important that we work as a community to recognise the critical importance of early diagnosis.”

Matt Fellows


Good to learn about approval of The FDA’s  first two therapies for the treatment of cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis with concern of patients.


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