Tafamidis for the Treatment of Transthyretin Amyloidosis

Lorenzini, Massimiliano; Elliott, Perry

doi:10.2217/fca-2018-0078

Cited by 5 publications

(4 citation statements)

References 62 publications

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“…The incidence of adverse events is similar in the tafamidis and placebo groups. 14 It stabilizes transthyretin tetramer by binding to the thyroxine-binding site of TTR tetramer and preventing dissociation of TTR tetramer into monomers, thus reducing TTR amyloid. 15 For the treatment of hereditary TTR amyloidosis, new therapies have been released.…”

Section: Discussionmentioning

confidence: 99%

Amiloidosis cardiaca con falla cardiaca aguda como presentación clínica

Arango-Moreno¹,

Duque-Ramírez²,

Medina-Medina³

et al. 2022

Cardiovasc Metab Sci

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La amiloidosis es una enfermedad de depósito, donde un precursor proteico específico se pliega inadecuadamente desde su estructura terciaria a una de forma más plana, generando depósitos multisistémicos que comprometen la función de múltiples órganos. Se realizó una búsqueda no sistemática de la literatura, encontrando múltiples reportes de amiloidosis cardiaca en población estadounidense y europea, pero no se encontraron reportes de caso de amiloidosis cardiaca como presentación aguda de falla cardiaca en paciente anciano en Latinoamérica. Se presenta el caso clínico de una paciente de edad avanzada, quien debuta con falla cardiaca aguda de novo donde se pudo evidenciar un compromiso cardiaco por depósitos amiloides. La sospecha clínica es fundamental, ya que el compromiso cardiovascular ha aumentado en frecuencia en los últimos años, y un diagnóstico oportuno muchas veces determina el pronóstico.

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Section: Discussionmentioning

confidence: 99%

Amiloidosis cardiaca con falla cardiaca aguda como presentación clínica

Arango-Moreno¹,

Duque-Ramírez²,

Medina-Medina³

et al. 2022

Cardiovasc Metab Sci

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show abstract

“…The patient was further referred for DNA analysis, which detected a Glu89Gln mutation in exon 3 of the TTR gene and the final diagnosis of ATTR was made. Treatment with the orphan drug tafamidis [7] was started and the patient currently shows no disease progression.…”

Section: Case Reportmentioning

confidence: 99%

Transthyretin Amyloidosis with Gastrointestinal Manifestation: a Case Report

Nakov

Sarafov

Nakov

et al. 2019

JGLD

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Transthyretin amyloidosis (ATTR) is a rare, progressive, life-threatening, hereditary disorder caused by mutations in the transthyretin gene. Due to the phenotypic heterogeneity, ATTR is difficult to recognize and it is often diagnosed very late. In ATTR gastrointestinal (GI) disorders play an important role in the patients’ morbidity and mortality. In some cases, GI symptoms are present even before the onset of the peripheral polyneuropathy. However, the complaints are various and it is really difficult to differentiate them from other GI disorders. We present a 61-year old male referred for diarrhea, unintentional weight loss and early satiety. He had hypotension after longstanding hypertension, numbness and tingling in the feet. We considered a broad differential diagnosis spectrum of chronic diarrhea syndrome and performed numerous laboratory, biochemical, imaging, endoscopic, histological and genetic tests. Transthyretin amyloidosis with a Glu89Gln mutation was diagnosed. Transthyretin amyloidosis is frequently misdiagnosed, representing a diagnostic challenge in GI practice. The presence of certain clinical combinations could help gastroenterologists to include ATTR in their diagnostic work-up.

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“…Tafamidis is a non‐nonsteroidal anti‐inflammatory benzoxazole derivative that binds with high affinity and selectivity to TTR, stabilizing the TTR tetramer, arresting the amyloid cascade, and disrupting the progression of ATTR amyloidosis 9,10 . In a pivotal 18‐month, randomized, placebo‐controlled trial, tafamidis was shown to reduce neurologic deterioration, preserve nerve function, and maintain quality of life in patients with early‐stage ATTR‐PN 11,12 .…”

mentioning

confidence: 99%

“…8 Tafamidis is a non-nonsteroidal anti-inflammatory benzoxazole derivative that binds with high affinity and selectivity to TTR, stabilizing the TTR tetramer, arresting the amyloid cascade, and disrupting the progression of ATTR amyloidosis. 9,10 In a pivotal 18-month, randomized, placebo-controlled trial, tafamidis was shown to reduce neurologic deterioration, preserve nerve function, and maintain quality of life in patients with early-stage ATTR-PN. 11,12 Evidence from the clinical development program, including open-label extension studies providing data from up to 6 years of treatment, and large registry and referral center studies, providing real-world data from up to 10 years, supports the longterm safety and effectiveness of tafamidis in delaying neurologic deterioration and prolonging survival in ATTR-PN.…”

mentioning

confidence: 99%

The Bioequivalence of Tafamidis 61‐mg Free Acid Capsules and Tafamidis Meglumine 4 × 20‐mg Capsules in Healthy Volunteers

Lockwood

O'Gorman

et al. 2020

Clinical Pharm in Drug Dev

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Tafamidis, a non-nonsteroidal anti-inflammatory benzoxazole derivative, acts as a transthyretin (TTR) stabilizer to slow progression of TTR amyloidosis (ATTR). Tafamidis meglumine, available as 20-mg capsules, is approved in more than 40 countries worldwide for the treatment of adults with early-stage symptomatic ATTR polyneuropathy. This agent, administered as an 80-mg, once-daily dose (4 × 20-mg capsules), is approved in the United States, Japan, Canada, and Brazil for the treatment of hereditary and wild-type ATTR cardiomyopathy in adults. An alternative single solid oral dosage formulation (tafamidis 61-mg free acid capsules) was developed and introduced for patient convenience (approved in the United States, United Arab Emirates, and European Union). In this single-center, open-label, randomized, 2-period, 2-sequence, crossover, multiple-dose phase 1 study, the rate and extent of absorption were compared between tafamidis 61-mg free acid capsules (test) and tafamidis meglumine 80-mg (4 × 20-mg) capsules (reference) after 7 days of repeated oral dosing under fasted conditions in 30 healthy volunteers. Ratios of adjusted geometric means (90%CI) for the test/reference formulations were 102.3 (98.0-106.8) for area under the concentration-time profile over the dosing interval and 94.1 (89.1-99.4) for the maximum observed concentration, satisfying prespecified bioequivalence acceptance criteria (90%CI, 80-125). Both tafamidis regimens had an acceptable safety/tolerability profile in this population.

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Tafamidis for the Treatment of Transthyretin Amyloidosis

Cited by 5 publications

References 62 publications

Amiloidosis cardiaca con falla cardiaca aguda como presentación clínica

Amiloidosis cardiaca con falla cardiaca aguda como presentación clínica

Transthyretin Amyloidosis with Gastrointestinal Manifestation: a Case Report

The Bioequivalence of Tafamidis 61‐mg Free Acid Capsules and Tafamidis Meglumine 4 × 20‐mg Capsules in Healthy Volunteers

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