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 and has a PDUFA action date set for August 11, 2018.
For our complete review of Patisiran:
Executive Summary of Evaluation:
|Patisiran (Alnylam Pharmaceuticals Inc.)|
|Orphan Drug Designation||6/14/2012|
|FDA Fast Track Designation||11/11/2013|
|Breakthrough Therapy Designation||11/20/2017|
|Priority Review Designation||2/1/2018|
|Indications||Familial Amyloid Neuropathies||Submitted for Approval (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 .
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-attr .
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.
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.