PFE - Despite FDA Doubts Adcomm Backs Alnylam's Gene Silencing Therapy For Expanded Use Into Rare Heart Disease | Benzinga
On Wednesday, an FDA advisory committee voted 9-3 in favor of Alnylam Pharmaceuticals Inc's (NASDAQ: ALNY) patisiran. It has a favorable benefit-risk profile in cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
ATTR is caused by misfolded transthyretin (TTR) proteins, accumulating as amyloid deposits in various body parts, including the heart, resulting in cardiomyopathy and heart failure.
Patisiran is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads ...