Posted by Michael Wonder on 06 May 2019
FDA approves new treatments for heart disease caused by a serious rare disease, transthyretin‑mediated amyloidosis
6 May 2019 - On May 3, the U.S. FDA approved Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) capsules for the treatment of the heart disease (cardiomyopathy) caused by transthyretin mediated amyloidosis in adults.
These are the first FDA-approved treatments for transthyretin mediated amyloidosis. Vyndaqel and Vyndamax have the same active moiety, tafamidis, but they are not substitutable on a milligram to milligram basis and their recommended doses differ.
The FDA granted Vyndaqel fast track, priority review and breakthrough therapy designations. Vyndaqel and Vyndamax each received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.
Approval of Vyndaqel and Vyndamax were granted to FoldRx, a subsidiary of Pfizer.
