FDA approves treatments for heart failure caused by rare disease

FDA approves treatments for heart failure caused by rare disease

(HealthDay)—Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules have been approved to treat adults with cardiomyopathy caused by transthyretin mediated amyloidosis (ATTR-CM), the U.S. Food and Drug Administration announced today.

Recommended dosage is four 20-mg capsules of Vyndaqel once daily or a single 61-mg capsule of Vyndamax once daily, according to the manufacturer. The two drugs, which are the first approved treatment for ATTR-CM, have the same moiety of tafamidis but are not substitutable on a per-milligram basis, the FDA said.

Approval was based on a clinical trial of 441 patients with ATTR-CM who were randomly assigned to receive Vyndaqel or placebo. Patients receiving Vyndaqel had a higher survival rate at an average of 30 months and a reduction in cardiovascular-related hospitalizations compared with receiving placebo.

Although patient numbers in the were small, researchers did not identify any drug-related side effects. The manufacturer's prescribing information for Vyndaqel lists potential side effects of diarrhea, , vaginal infection, and abdominal pain. Because of the risk for fetal harm with tafamidis, the FDA recommends providers discuss pregnancy planning and prevention with women.

Approval was granted to FoldRx, a subsidiary of Pfizer.

More information: More Information

Copyright © 2019 HealthDay. All rights reserved.

Citation: FDA approves treatments for heart failure caused by rare disease (2019, May 7) retrieved 13 July 2024 from https://medicalxpress.com/news/2019-05-fda-treatments-heart-failure-rare.html
This document is subject to copyright. Apart from any fair dealing for the purpose of private study or research, no part may be reproduced without the written permission. The content is provided for information purposes only.

Explore further

Drug reduces deaths and hospitalizations from underdiagnosed form of heart failure


Feedback to editors