Drugs List

Therapeutic Indications

Uses

Treatment of wild-type or hereditary ATTR-CM

Treatment of wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy.

Contraindications

Children under 18 years
Solid organ transplant recipients
Breastfeeding
Hereditary fructose intolerance
Pregnancy

Precautions and Warnings

Females of childbearing potential
New York Heart Association class IV failure
Renal impairment - creatinine clearance below or equal to 30ml/minute
Severe hepatic impairment

Treatment to be initiated and supervised by a specialist
Presentations with sorbitol unsuitable in hereditary fructose intolerance
May affect results of some laboratory tests
Discontinue if patient undergoes organ transplant
Female: Contraception required during and for 1 month after treatment

Patients should be monitored continually to assess the need for other therapy, including the need for organ transplantation.

Before treatment begins with 61mg dose, ensure patients presenting with specific medical history or signs of heart failure or cardiomyopathy have an aetiologic diagnosis to confirm transthyretin amyloidosis with cardiomyopathy and exclude AL amyloidosis. Diagnosis should be performed via bone scintigraphy and blood/urine assessment and/or by biopsy, and transthyretin genotyping to characterise as wild type or hereditary.

Treatment should be initiated as soon as possible in the disease course. However if amyloid related cardiac damage is advanced such as New York Heart Association Class III, treatment initiation or continuation is taken at the discretion of the physician.

Pregnancy and Lactation

Pregnancy

Tafamidis is contraindicated during pregnancy.

The manufacturers do not recommend using tafamidis during pregnancy and in women of childbearing potential not using contraception. Animal studies have shown developmental toxicity. Human data is limited and as such a potential risk cannot be ruled out.

Lactation

Tafamidis is contraindicated during breastfeeding.

The manufacturers do not recommend breastfeeding whilst taking tafamidis. Animal studies indicate tafamidis is expressed in the breast milk, but the quantity is unknown.

Side Effects

Altered liver function tests
Flatulence

Presentation

Oral formulations of tafamidis.

Drugs List

Therapeutic Indications

Uses

Treatment of wild-type or hereditary ATTR-CM

Treatment of wild type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy.

Dosage

Tafamidis and tafamidis meglumine are not interchangeable on a per mg basis.

Adults

Additional Dosage Information

Contraindications

Children under 18 years
Solid organ transplant recipients
Breastfeeding
Hereditary fructose intolerance
Pregnancy

Precautions and Warnings

Females of childbearing potential
New York Heart Association class IV failure
Renal impairment - creatinine clearance below or equal to 30ml/minute
Severe hepatic impairment

Treatment to be initiated and supervised by a specialist
Presentations with sorbitol unsuitable in hereditary fructose intolerance
May affect results of some laboratory tests
Discontinue if patient undergoes organ transplant
Female: Contraception required during and for 1 month after treatment

Patients should be monitored continually to assess the need for other therapy, including the need for organ transplantation.

Before treatment begins with 61mg dose, ensure patients presenting with specific medical history or signs of heart failure or cardiomyopathy have an aetiologic diagnosis to confirm transthyretin amyloidosis with cardiomyopathy and exclude AL amyloidosis. Diagnosis should be performed via bone scintigraphy and blood/urine assessment and/or by biopsy, and transthyretin genotyping to characterise as wild type or hereditary.

Treatment should be initiated as soon as possible in the disease course. However if amyloid related cardiac damage is advanced such as New York Heart Association Class III, treatment initiation or continuation is taken at the discretion of the physician.

Pregnancy and Lactation

Pregnancy

Tafamidis is contraindicated during pregnancy.

The manufacturers do not recommend using tafamidis during pregnancy and in women of childbearing potential not using contraception. Animal studies have shown developmental toxicity. Human data is limited and as such a potential risk cannot be ruled out.

Lactation

Tafamidis is contraindicated during breastfeeding.

The manufacturers do not recommend breastfeeding whilst taking tafamidis. Animal studies indicate tafamidis is expressed in the breast milk, but the quantity is unknown.

Side Effects

Altered liver function tests
Flatulence

Effects on Laboratory Tests

An increase in liver function tests and a decrease in total thyroxine with no accompanying change to free thyroxine (T4) or thyroid stimulating hormone (TSH) may occur during tafamidis treatment.

Overdosage

It is strongly recommended that the UK National Poisons Information Service be consulted on cases of suspected or actual overdose where there is doubt over the degree of risk or about appropriate management.

The following number will direct the caller to the relevant local centre (0844) 892 0111

Information may be obtained if you have access to ToxBase the primary clinical toxicology database of the National Poisons Information Service. This is available via password on the internet ( www.toxbase.org ) or if this is unavailable at the backup site ( www.toxbasebackup.org ).

Further Information

Last Full Review Date: September 2020

Reference Sources

Summary of Product Characteristics: Vyndaqel 61mg soft capsules. Pfizer Ltd. Revised February 2020.