‘Gene Silencing’ Drug Under FDA Review for Hereditary ATTR Amyloidosis

Pexidartinib is an oral small molecule that is believed to work by inhibiting colony stimulating factor-1 receptor (CSF1R).
Patisiran has been granted Fast Track Designation, Breakthrough Therapy Designation, and an expanded Orphan Drug Designation for ATTR amyloidosis from the FDA.

Alnylam announced that the Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for patisiran, an investigational drug for the treatment of hereditary aTTR amyloidosis, for Priority Review.

Hereditary aTTR amyloidosis is an inherited, progressively debilitating, and often life-threatening disease caused by mutations in the TTR gene. Mutations result in an accumulation of abnormal amyloid proteins which damage organs and tissue, causing intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy.

Patisiran is an intravenous RNA interference (RNAi) therapeutic agent that targets transthyretin (TTR). Its design silences specific messenger RNA, potentially inhibiting the production of TTR protein before it is made. Blocking production may help enable the clearance of TTR amyloid deposits in the peripheral tissues and potentially restore function to these tissues. 

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Patisiran has been evaluated in several Phase 2 studies, open-label extension studies, an expanded access protocol study, and the APOLLO study. The target Prescription Drug User Fee Act (PDUFA) date has been set for August 11, 2018. 

For more information call (866) 330-0326 or visit Alynylam.com.