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Alnylam Pharmaceuticals to Present New Drug Application for Cardiomyopathy of ATTR Amyloidosis

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Alnylam Pharmaceuticals Inc. announced on Wednesday that its stock will be temporarily halted on Nasdaq while a regulatory advisory committee reviews its new drug application for the treatment of Cardiomyopathy of ATTR Amyloidosis. The treatment, known as onpattro or patisiran, has already received approval in the United States and Canada for the treatment of polyneuropathy of hereditary ATTR (hATTR) amyloidosis in adults.

ATTR amyloidosis is a severe and life-threatening disease that is characterized by the accumulation of misfolded transthyretin (TTR) proteins in different parts of the body, including the nerves, heart, and gastrointestinal tract. Patients with ATTR amyloidosis can experience either polyneuropathy, cardiomyopathy, or both manifestations of the disease.

Alnylam reports that there are two forms of ATTR amyloidosis: hereditary ATTR (hATTR) amyloidosis, which is caused by a TTR gene variant and affects approximately 50,000 individuals worldwide, and wild-type ATTR (wtATTR) amyloidosis, which occurs without a TTR gene variant and impacts an estimated 200,000 to 300,000 people globally.

The FDA advisory panel will convene at 9:00 a.m. ET to discuss the new drug application. The FDA has set an action date for the application on October 8.

Alnylam’s stock performance has experienced an 11% decline year-to-date, in contrast to the S&P 500’s 16.2% gain.

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