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The U.S. Food and Drug Administration (FDA) recently approved Pfizer's drug, VYNDAQEL and chlorobenzene (VYNDAMAX), to reduce cardiovascular-related mortality and hospitalization rates for the use of wild or genetic transthyroidin amylotype cardiomyopathy (ATTR-CM) in adults, according to Pfizer's official website.
VYNDAQEL and VYNDAMAX are the first two oral formulations of the thyroxine protein stabilizer chlorobenzene, and the first and currently the only FDA-approved treatment for ATTR-CM.
thyroxine protein amyloid myopathy is a rare, deadly heart disease characterized by the misfolded protein formation abnormal substance (amyloid) deposited in the heart, manifested as restrictive cardiomyopathy and progressive heart failure. In the past, there were no approved drugs, and available treatment options only included treatment for the disease, even heart (or heart and liver) transplants in individual cases. It is estimated that there are currently about 100,000 patients in the United States, of whom only 1-2% are diagnosed. Globally,
VYNDAQEL and VYNDAMAX have the same active part of tafamidis, but they cannot be replaced with the same milligram stake in use, and their recommended doses are different. The recommended dose of VYNDAQEL is 80 mg (4 capsules of 20 mg), taken orally once daily. The recommended dose of VYNDAMAX is 61 mg (1 capsule) per session, taken orally, once daily. VYNDAQEL and VYNDAMAX are both designated as "orphan drugs" by the FDA.
VYNDAQEL obtained the orphan drug for treatment at ATTR-CM in the European Union in 2012 and the orphan drug in Japan in 2018. In November 2018, the FDA approved the VYNDAQEL New Drug Application (NDA) priority review eligibility. In March 2019, the Ministry of Health, Labour and Welfare of Japan approved the award of VYNDAQEL for the sAKIGAKE (leading, pioneering) drug designation for the treatment of wild and mutant ATTR-CM patients. According to Reuters, VYNDAQEL is expected to price $225,000 a year.
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