Vyndaqel

[Introduction to Vyndaqel]

Recently, the U.S. Food and Drug Administration (FDA) has approved VYNDAQEL (tafamidis meglumine) and VYNDAMAX (tafamidis) for the treatment of adults with wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM) to reduce cardiovascular-related mortality and hospitalization rates.

VYNDAQEL and VYNDAMAX are the two oral formulations of the first transthyretin protein stabilizer clofenac. They are also the first and currently the only drug approved by the FDA for the treatment of ATTR-CM.

Transthyretin amyloid cardiomyopathy is a rare and fatal disease characterized by the deposition of abnormal misfolded proteins (amyloid) in the heart, manifesting as restrictive cardiomyopathy and progressive heart failure. There are no drugs currently approved to treat ATTR-CM; available treatment options include only symptomatic treatment and, in individual cases, heart (or heart and liver) transplantation. There are currently approximately 100,000 ATTR-CM patients in the United States, of which only 1-2% are diagnosed.

Approval date: May 6, 2019 Company: Pfizer

YNDAQEL (tafamidis meglumine) capsules, for oral use

Initial U.S. approval: 2019

VYNDAMAX TM (tafamidis) capsules for oral administration

Initial US approval: 2019

[Mechanism of action of Vyndaqel]

Tafamidis is a selective stabilizer of TTR. Tafamidis binds to TTR at the thyroxine binding site, stabilizing the compound and slowing dissociation into monomers, the rate-limiting step in amyloid formation.

[Vyndaqel Indications and Usage]

VYNDAQEL and VYNDAMAX are transthyretin protein stabilizers, used to treat wild-type or hereditary cardiomyopathy

Transthyretin-mediated amyloidosis in adults can reduce cardiovascular mortality and cardiovascular-related hospitalization rates.

[Vyndaqel dosage and dosage]

The recommended dosage is:

VYNDAQEL 80 mg orally per day, or

VYNDAMAX 61 mg orally per day

VYNDAMAX and VYNDAQEL cannot be substituted on a per mg basis.

[Vyndaqel dosage form and strength]

Capsules: Tafamidis meglumine 20mg and tafamidis 61mg.

[Contraindications of Vyndaqel]

None.

[Vyndaqel is used in specific populations]

Pregnancy: Based on animal studies, it may cause fetal harm.

Lactation period: It is recommended not to breastfeed.

[Vyndaqel Packaging Provision/Storage and Handling]

VYNDAQEL 20 mg (tafamidis meglumine) soft gelatin capsules are yellow, opaque, oval-shaped, and printed with "VYN 20".

VYNDAQEL Capsules

Specifications

20 mg NDC: 6609-1975-40

Pack configuration

Carton with 4 middle cartons. Each intermediate carton contains 3 blister cards. Each blister card contains 10 capsules. (120 capsules in total)

VYNDAMAX 61 mg (tafamidis) soft gelatin capsules are reddish-brown, opaque, oblong, and printed

VYNDAMAX CAPSULES

Specifications

61 mg NDC: 6609-8730-30

Pack configuration

3 blister cards in carton. Each blister card contains 10 capsules. (30 capsules total)

Store VYNDAQEL and VYNDAMAX at controlled room temperature, 20°C to 25°C (68°F to 77°F); permitted temperature is 15°C to 30°C (59°F to 86°F)