FDA OKs Two Tafamidis Formulations for Rare Cardiomyopathy

Patrice Wendling

May 06, 2019

The US Food and Drug Administration (FDA) has approved Pfizer's tafamidis meglumine (Vyndaqel ) and tafamidis (Vyndamax) capsules to treat transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM) in adults.

These are the first FDA-approved treatments for ATTR-CM, a rare and often fatal disease caused by deposition of amyloid fibrils in the myocardium.

"The treatments we're approving today are an important advancement in the treatment of the cardiomyopathy caused by transthyretin-mediated amyloidosis," Norman Stockbridge, MD, PhD, director of the Division of Cardiovascular and Renal Drugs in the FDA's Center for Drug Evaluation and Research, said in a news release.

The drugs are not substitutable on a per milligram basis, and their recommended doses differ. The recommended dosage is either Vyndaqel 80 mg orally once-daily, taken as four 20 mg capsules, or Vyndamax 61 mg orally once-daily, taken as a single capsule.

The approvals for both drugs are based on the multicenter ATTR-ACT trial in 441 patients with ATTR-CM. After 30 months follow-up, Vyndaqel was associated with a reduction in all-cause mortality and cardiovascular hospitalizations compared with placebo.

The number of patients in clinical studies was small, but no drug-associated side effects have been identified, the FDA says.

However, tafamidis may cause fetal harm when administered to a pregnant woman. Women taking Vyndaqel or Vyndamax should discuss pregnancy planning and prevention with their healthcare professional.

"ATTR-CM is not only fatal, but also significantly underdiagnosed, with some patients cycling through multiple doctors and a myriad of tests over a period of years while the disease progresses," Isabelle Lousada, founder and CEO of the Amyloidosis Research Consortium, said in a statement. "ATTR-CM is a rare disease for which more education and awareness is needed. The approval of these medicines represents an important advance for patients; however, it is equally important that we work as a community to recognize the critical importance of early diagnosis."

Vyndaqel and Vyndamax each received orphan drug designation from the FDA. Approval of Vyndaqel and Vyndamax were granted to FoldRx, a subsidiary of Pfizer.

The list price for Vyndaqel is expected to be $225,000 a year and analysts are estimating annual sales to exceed $1 billion in 2024, according to Reuters.

Follow Patrice Wendling on Twitter: @pwendl. For more from theheart.org | Medscape Cardiology, follow us on Twitter and Facebook

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