Tegsedi™ (Inotersen sodium) – 2018 FDA Approval

FDA Approval: 10/5/2018

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, developed by Ionis Pharmaceuticals Inc., in collaboration with Akcea Therapeutics, for the treatment of FAP was approved by the FDA on October 5, 2018.  Inotersen had previously been 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 (Tegsedi™):


Executive Summary of Evaluation:

    Inotersen sodium (Ionis Pharmaceuticals Inc.)


Status Marketed
PDUFA 10/6/2018
  Orphan Drug Designation 7/24/2012
  FDA Fast Track Designation 12/13/2012
  Priority Review Designation 1/8/2018
  FDA Approval 10/5/2018
  Patent Expiration 4/29/2031
Indications Familial Amyloid Neuropathies Approval (NDA)
Efficacy Familial Amyloid Neuropathies Efficacy reported in Phase III Trials



Akcea Therapeitics Press Releases “Akcea and Ionis Receive FDA Approval of TEGSEDI™ (inotersen) for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults” https://ir.akceatx.com/news-releases/news-release-details/akcea-and-ionis-receive-fda-approval-tegseditm-inotersen Accessed October 16, 2018

“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, investors.alnylam.com/news-releases/news-release-details/alnylam-announces-fda-acceptance-new-drug-application-nda-and.

“A Rare Disease with a Global Presence.” Recognizing TTR-FAP, www.recognizingttr-fap.com/epidemiology. Accessed: April 17, 2018.

Clinical Trial data obtained at https://clinicaltrials.gov/ct2/home Accessed: May 30, 2018.

Elvidge, Suzanne. “Ionis Defies Alnylam as It Drives Inotersen towards the Market.” BioPharma Dive, 8 Nov. 2017, www.biopharmadive.com/news/ionis-defies-alnylam-as-it-drives-inotersen-towards-the-market/510384/.

“Ionis Presents New Data from NEURO-TTR Study at European ATTR Amyloidosis Meeting.” Ionis Pharmaceuticals, Inc., 2 Nov. 2017, ir.ionispharma.com/news-releases/news-release-details/ionis-presents-new-data-neuro-ttr-study-european-attr.

Pharma Intelligence Center https://pharma.globaldata.com Accessed: May 30, 2018.

U.S. Food & Drug Administration Drugs@FDA: FDA Approved Drug Products Product Labeling https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/211172lbl.pdf Accessed: October 16, 2018


Additional 2018 Pipeline Reviews are available in our pipeline library.


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.