Inotersen sodium – 2018 Pipeline Review

Familial amyloid polyneuropathy (FAP, also called transthyretin amyloidosis) is a hereditary neurodegenerative disease that is characterized by the accumulation and deposition of the transthyretin protein (TTR) in the tissues and peripheral nervous system, and often causes pain, muscle weakness, and autonomic dysfunction. Eventually, the disease progresses into a sensory and motor polyneuropathy. Currently, the mainstay of treatment for FAP is liver transplantation which removes the source of abnormal TTR. Symptomatic management focuses on reducing neuropathic pain and improving gastrointestinal and other autonomic symptoms.

Inotersen sodium is an investigational drug candidate being developed by Ionis Pharmaceuticals Inc., in collaboration with Akcea Therapeutics, for the treatment of FAP.  Inotersen was granted Priority Review of its NDA by the FDA on January 8, 2018 for its indication of Familial Amyloid Neuropathies, and originally had an anticipated PDUFA date of July 6, 2018. In early May, the FDA changed the action date to October 6, 2018.


For our complete review of Inotersen sodium:



Executive Summary of Evaluation:


Inotersen sodium (Ionis Pharmaceuticals Inc.)







  Orphan Drug Designation


  FDA Fast Track Designation


  Priority Review Designation


  Patent Expiration


Indications Familial Amyloid Neuropathies

Submitted for Approval (NDA)

Efficacy Familial Amyloid Neuropathies

Efficacy reported in Phase III Trials



“Alnylam Announces FDA Acceptance of New Drug Application (NDA) and Priority Review Status for Patisiran, an Investigational RNAi Therapeutic for the Treatment of Hereditary ATTR (HATTR) Amyloidosis.” Alnylam Pharmaceuticals, Inc., 1 Feb. 2018,

“A Rare Disease with a Global Presence.” Recognizing TTR-FAP, Accessed: April 17, 2018.

Clinical Trial data obtained at Accessed: May 30, 2018.

“Ionis Presents New Data from NEURO-TTR Study at European ATTR Amyloidosis Meeting.” Ionis Pharmaceuticals, Inc., 2 Nov. 2017,

Pharma Intelligence Center Accessed: May 30, 2018.

Elvidge, Suzanne. “Ionis Defies Alnylam as It Drives Inotersen towards the Market.” BioPharma Dive, 8 Nov. 2017,

 Additional 2018 Pipeline Reviews are available for the following drugs.




Coauthored by: Kristen Ciampi, PharmD

Kristen recently graduated with a PharmD degree from the University of Rhode Island in May of 2018, and is a Pharmacy Analytics Intern at RJ Health Systems. 

For our complete review of Inotersen sodium: