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Tafamidis, sold under the brand names Vyndaqel and Vyndamax, [5] is a medication used to delay disease progression in adults with certain forms of transthyretin amyloidosis. It can be used to treat both hereditary forms, familial amyloid cardiomyopathy and familial amyloid polyneuropathy , as well as wild-type transthyretin amyloidosis , which ...
In late 2011, the European Medicines Agency approved the transthyretin kinetic stabilizer Tafamidis or Vyndaqel discovered by Jeffery W. Kelly and developed by FoldRx pharmaceuticals (acquired by Pfizer in 2010) for the treatment of FAP based on clinical trial data. Tafamidis (20 mg once daily) slowed the progression of FAP over a 36-month ...
Pfizer Inc said on Monday that its drug tafamidis reduced the risk of death for patients with a rare and fatal heart disease by around 30 percent, boosting the prospects of what could be a billion ...
(Reuters) -Alnylam Pharmaceuticals said on Monday it would not pursue expanded use of its drug to treat a potentially fatal heart disease in the U.S. after the Food and Drug Administration ...
ALN-TTR02 recently received orphan drug status in the U.S. and the EU and will start phase 3 study by late 2013. ALN-TTRsc will complete phase 2 study by late 2013 and start phase 3 study by 2014 ...
Priority review vouchers are currently earned by pharmaceutical companies for the development and approval of drugs treating neglected tropical diseases, rare pediatric diseases, and "medical countermeasures" for terrorism. The voucher can be used for future drugs that could have wider indications for use, but the company is required to pay a ...
In another key category, our Vyndaqel family of products achieved continuing momentum with 90% year-over-year growth in the U.S. and 32% operational growth in international markets.
They estimate a market of 35,000 patients in the U.S. and Europe, with an additional 23,000 in some countries in the Middle East where Casgevy is also approved.