Disease‐modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications
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Wiley
2024
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| author | Erika L. Hellenbart Heather J. Ipema Mary C. Rodriguez‐Ziccardi Hema Krishna Robert J. DiDomenico |
| author_facet | Erika L. Hellenbart Heather J. Ipema Mary C. Rodriguez‐Ziccardi Hema Krishna Robert J. DiDomenico Erika L. Hellenbart Heather J. Ipema Mary C. Rodriguez‐Ziccardi Hema Krishna Robert J. DiDomenico |
| collection | Wiley Open Access |
| contents | Disease‐modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications Erika L. Hellenbart Heather J. Ipema Mary C. Rodriguez‐Ziccardi Hema Krishna Robert J. DiDomenico Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy Abstract Transthyretin amyloidosis (ATTR) is a rare disease that results in amyloid fibril misfolding and deposition in multiple organs, including the heart, leading to the development of ATTR cardiomyopathy (ATTR‐CM), which is associated with poor outcomes. In the last decade, several disease‐modifying medications are in advanced stages of clinical development or have been approved to treat ATTR‐CM. The purpose of this review is to critically evaluate clinical trial data investigating the use of approved and investigational medications for the treatment of ATTR‐CM. We performed a comprehensive literature search via PubMed and EMBASE to identify randomized controlled trials evaluating medications for the treatment of ATTR‐CM published through August 2024. This narrative review describes the pathophysiology of ATTR‐CM, highlights important screening and diagnostic work‐up, and summarizes the existing clinical evidence resulting from our literature search. Several classes of disease‐modifying medications are in development for ATTR‐CM. The tetramer stabilizers and transthyretin silencers have proven to be the most effective therapies to date. Tafamidis and acoramidis are currently approved for ATTR‐CM while vutrisiran approval for ATTR‐CM may be forthcoming. Other disease‐modifying medication classes in development include antisense oligonucleotides, gene editing therapies, and monoclonal antibodies. However, several unmet needs exist including the lack of cost‐effectiveness due to the extremely high acquisition costs of these medications. Disease‐modifying medications approved and in development to treat ATTR‐CM offer hope for patients with this disease, but their lack of affordability is the biggest barrier to their use. 10.1002/phar.4639 http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| doi_str_mv | 10.1002/phar.4639 |
| format | Artículo Open Access |
| id | wiley_oa_10_1002_phar_4639 |
| institution | Wiley Open Access |
| license_str_mv | http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| publishDate | 2024 |
| publisher | Wiley |
| record_format | wiley_oa |
| spellingShingle | Disease‐modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications Erika L. Hellenbart Heather J. Ipema Mary C. Rodriguez‐Ziccardi Hema Krishna Robert J. DiDomenico Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy Disease‐modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications Erika L. Hellenbart Heather J. Ipema Mary C. Rodriguez‐Ziccardi Hema Krishna Robert J. DiDomenico Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy Abstract Transthyretin amyloidosis (ATTR) is a rare disease that results in amyloid fibril misfolding and deposition in multiple organs, including the heart, leading to the development of ATTR cardiomyopathy (ATTR‐CM), which is associated with poor outcomes. In the last decade, several disease‐modifying medications are in advanced stages of clinical development or have been approved to treat ATTR‐CM. The purpose of this review is to critically evaluate clinical trial data investigating the use of approved and investigational medications for the treatment of ATTR‐CM. We performed a comprehensive literature search via PubMed and EMBASE to identify randomized controlled trials evaluating medications for the treatment of ATTR‐CM published through August 2024. This narrative review describes the pathophysiology of ATTR‐CM, highlights important screening and diagnostic work‐up, and summarizes the existing clinical evidence resulting from our literature search. Several classes of disease‐modifying medications are in development for ATTR‐CM. The tetramer stabilizers and transthyretin silencers have proven to be the most effective therapies to date. Tafamidis and acoramidis are currently approved for ATTR‐CM while vutrisiran approval for ATTR‐CM may be forthcoming. Other disease‐modifying medication classes in development include antisense oligonucleotides, gene editing therapies, and monoclonal antibodies. However, several unmet needs exist including the lack of cost‐effectiveness due to the extremely high acquisition costs of these medications. Disease‐modifying medications approved and in development to treat ATTR‐CM offer hope for patients with this disease, but their lack of affordability is the biggest barrier to their use. 10.1002/phar.4639 http://creativecommons.org/licenses/by-nc-nd/4.0/ |
| title | Disease‐modifying therapies for amyloid transthyretin cardiomyopathy: Current and emerging medications |
| topic | Pharmacotherapy: The Journal of Human Pharmacology and Drug Therapy |
| url | https://accpjournals.onlinelibrary.wiley.com/doi/10.1002/phar.4639 |