Alnylam Pharmaceuticals Inc. ALNY said Wednesday its stock will be halted on Nasdaq through the day as a regulatory advisory committee will meet to review its new drug application for a treatment for the Cardiomyopathy of ATTR Amyloidosis. The treatment called onpattro, or patisiran, is approved in the U.S. and Canada for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults. ATTR is a progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients can have polyneuropathy, cardiomyopathy, or both manifestations of the disease. There are two different forms of ATTR amyloidosis — hereditary ATTR (hATTR) amyloidosis, which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR) amyloidosis, which occurs without a TTR gene variant and impacts an estimated 200,000 — 300,000 people worldwide, according to Alnylam. The FDA advisory panel will meet at 9:00 a.m. ET. The FDA has set an Oct. 8 action date for the application. Alnylam’s stock is down 11% in the year to date, while the S&P 500 SPX has gained 16.2%.
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