Researchers reported that tafamidis is efficacious in securing better outcomes for patients with hereditary amyloid transthyretin amyloidosis (hATTR), in a study published in Drug, Healthcare and Patient Safety. 

Researchers have been working to improve the therapeutic landscape of hATTR. Major developments, such as the introduction of disease-modifying therapies, have helped patients achieve important treatment milestones, but liver transplantation remains the only way to eliminate the production of amyloidogenic mutated TTR by the liver. 

“Considering the pathogenic TTR pathway, stabilizing the mutant TTR protein, and then preventing its dissociation and consequent amyloid fibril formation, would be an optimal target for newer agents,” the authors of the study said.

Tafamidis, an oral TTR stabilizer, has been thoroughly investigated for these very properties. It was developed during the early stages of the disease and soon gained regulatory approval across the globe. It is now the first-line treatment for early hATTR in more than 40 countries, Falcão de Campos and Conceição, the authors of the current review, wrote.

The authors of the study sought to perform a literature review appraising the therapy in terms of its safety and efficacy. Their study invites readers to go on a journey in tracking the progress of tafamidis through the years and examine how it is impacting care today.

Read more about hATTR etiology 

The authors found that the efficacy of tafamidis has been validated in a number of early-stage hATTR studies. One study used data from real-world clinical usage of tafamidis. The verdict? When tafamidis is used over time, it delays disease progression, primarily neuropathy severity. This then decreases the likelihood of needing further treatment.

However, the authors of the review recognized that the use of tafamidis alone would not resolve all the issues arising from a multisystemic disease such as hATTR. From the get-go, they were firm in their convictions for the need to set up a multidisciplinary team that can handle various aspects of the disease.

“An experienced and multidisciplinary team, including a neurologist and cardiologist, should regularly and carefully monitor all treated patients in order to assess response to treatment and address any adverse events,” they wrote.

hATTR primarily drives pathology in the nervous systems (both peripheral and autonomic) and the heart in affected patients. It is characterized by extracellular depositions of amyloid fibrils in various organs in the body; left untreated, patients usually have an estimated life expectancy range of a decade.

Liver transplantation in these patients is extremely risky, and candidates likely have to wait a considerable amount of time before finding donor compatibility. Patients who have had this procedure may still experience disease progression, particularly when amyloid fibril production has already begun in the eye pre-surgery, which can lead to severe eye disease. 


Falcão de Campos C, Conceição I. Updated evaluation of the safety, efficacy and tolerability of tafamidis in the treatment of hereditary transthyretin amyloid polyneuropathyDrug Healthc Patient Saf. 2023;15:51-62. doi:10.2147/DHPS.S338577