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This represents the first FDA approved treatment in ATTR-CM for both the wild-type and hereditary forms of ATTR amyloidosis.

Vyndaqel/Vyndamax is a small molecule that binds to and stabilizes transthyretin (TTR), the culprit protein in ATTR amyloidosis. By acting as a TTR stabilizer, Vyndaqel/Vyndamax prevent TTR from misfolding and thus reduce the formation of amyloid fibrils that cause organ damage in ATTR.

FDA approval is based on results from the Phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) clinical trial. This trial evaluated the efficacy, safety, and tolerability of Vyndaqel, at both 20 and 80 mg doses, compared to placebo for the treatment of ATTR cardiomyopathy (ATTR-CM).

This FDA approval of Vyndaqel and Vyndamax comes on the heels of last year’s approvals of Onpattro (patisiran) and Tegsedi (inotersen), both approved for the treatment of hereditary ATTR polyneuropathy (hATTR-PN). Vyndaqel has been approved and used to treat hATTR-PN in more than 40 countries outside the US, including Japan, Mexico, Argentina, and across Europe. However, none of these approvals included patients with wildtype ATTR or hereditary ATTR cardiomyopathy.

“This is an epic moment for the treatment of ATTR amyloidosis. We are very excited and hopeful for the expanding landscape of ATTR amyloidosis by the approval of tafamidis, the first drug approved for ATTR cardiomyopathy.” Says Dr. Vaishali Sanchorawala, Director of the Amyloidosis Center at Boston University School of Medicine and Boston Medical Center, “Wildtype ATTR amyloidosis patients are being diagnosed now more than ever, and the approval of tafamidis represents a major success for this population.”

View more information about Vyndaqel and Vyndamax here>>

Read more about hereditary ATTR amyloidosis here>>

Read more about wild-type ATTR amyloidosis here>>

Read original press release from the FDA here>>