What are Vyndamax and Vyndaqel, and what do they treat?
Both contain tafamidis, a drug that stabilises the TTR protein to prevent amyloid deposits — approved in 2019 with orphan drug designation for a disease that kills within about 2.5 years if untreated.
Both contain tafamidis, a drug that stabilises the transthyretin (TTR) protein so it is less likely to misfold into amyloid deposits that stiffen the heart. Vyndaqel (tafamidis meglumine) and Vyndamax (tafamidis) were approved by the FDA in 2019 with orphan drug designation. ATTR-CM leads to heart failure and death, typically within about 2.5 years if untreated, and BridgeBio estimates there are around 240,000 US patients, many undiagnosed. Pfizer had this market entirely to itself for five years, which is central to many of the criticisms that followed.