FDA approves Pfizer Vyndaqel and Vyndamax for treatment of myocardial disease caused by amyloid degeneration

AATR-CM is a rare, life-threatening diseaseIt is caused by the instability of the TTRTTR usually exists in the form of tetrapolymers, which, when unstable tetrapolymers are dissofarted, cause setrusicleing errors in TTR aggregation and form amyloid progenitor fibers and amyloid protein depositionThe deposition of these amyloids in the heart can cause the heart muscle to harden and eventually cause heart failuretoday, Pfizer(http://announced that the U.Stheof the U.SFda (http://approved the listing of the company's Vyndaqel and Vyndamax to treat myocardial disease (ATTR-CM) caused by wild or hereditary thyroxineprotein-mediated amyloid diseaseAbout Vyndaqel and Vyndamax
Vyndaqel and Vyndamax are two oral formulations of the "first-in-class" transthyroxineprotein (TTR) stabilizer tafamidisIt is worth noting that this is the first FDA-approved treatment for AATR-CMVyndaqel and Vyndamax are oral TTR stabilizers that stabilize the tetrapolymer morphology of TTR by binding to TTR specifically, thus delaying the production of amyloid protein deposition leading to ATTR-CMVyndaqel has been awarded fast-track qualifications and breakthrough therapies by the FDAIt is seen by Pfizer as a potential lyingdrug(http://The studythis approval is based on the results of a key Phase 3 clinicaltrial(http://called AATR-ACTThis is a global clinical trial with a randomized double-blind, placebo-controlled, firsttesting(http://treatment of AATR-CMThe AATR-ACT trial results showed that Vyndaqel significantly reduced the combined index of all-cause mortality and cardiovascular-related hospitalization rates (p-0.0006) compared to placebo during the 30-month trialIn terms of safety
Vyndaqel not only reduced the risk of all-cause death by 30% (p-0.026) and reduced the risk of cardiovascular-related hospitalization by 32% (p0.0001), but also improved other functional indicators in patientsFor example, six months after treatment, Vyndaqel can reduce the decline in the patient's performance in the 6-minute walk test