Onpattro™ (Patisiran) – 2018 FDA Approval

FDA Approval: 8/10/2018

Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited and debilitating disease that is characterized by mutations in the TTR gene. These gene mutations can cause accumulation of abnormal amyloid proteins in body organs and tissue, leading to damage of these areas, such as the peripheral nerves. Peripheral, autonomic, or cardiomyopathy can result from this accumulation and damage, making this a progressive and potentially fatal disease. Currently, liver transplantation is the only available treatment option for hATTR amyloidosis.

Alnylam Pharmaceuticals has utilized RNAi technology to develop patisiran, an investigational antisense RNAi oligonucleotide, for the treatment of hATTR amyloidosis (Familial Amyloid Neuropathies). Patisiran received Priority Review designation by the FDA on February 1, 2018 with a PDUFA action date of August 11, 2018. FDA approval was granted on August 10, 2018.

For our complete review of Patisiran (Onpattro™):

Executive Summary of Evaluation:


Patisiran (Alnylam Pharmaceuticals Inc.)







  Orphan Drug Designation


  FDA Fast Track Designation


  Breakthrough Therapy Designation


  Priority Review Designation


  FDA Approval


  Patent Expiration


Indications Familial Amyloid Neuropathies

Approved (NDA)

Efficacy Familial Amyloid Neuropathies

Efficacy reported in Phase III Trials



“Alnylam Presents New Clinical Results from the APOLLO Phase 3 Study of Patisiran at the 16th International Symposium on Amyloidosis.” Alnylam Pharmaceuticals, Inc., 28 Mar. 2018, investors.alnylam.com/news-releases/news-release-details/alnylam-presents-new-clinical-results-apollo-phase-3-study Accessed: August 13, 2018.

Clinical Trial data obtained at https://clinicaltrials.gov/ct2/home, Accessed: June 12, 2018.

“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-att Accessed: August 13, 2018.

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

“A Rare Disease with a Global Presence.” Recognizing TTR-FAP, www.recognizingttr-fap.com/epidemiology . Accessed: June 13, 2018.

U.S. Food & Drug Administration, FDA News & Events, FDA News Release https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm616518.htm   Accessed: August 13, 2018

Additional 2018 Pipeline Reviews are available for the following drugs.

Moxetumomab pasudotox


Please see the July 2018 FDA Actions Newsletter for additional information on recent FDA approvals

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.