Inotersen
- Generic Name
- Inotersen
- Brand Names
- Tegsedi
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1492984-65-2
- Unique Ingredient Identifier
- 0IEO0F56LV
- Background
Inotersen is a transthyretin-directed antisense oligonucleotide for the treatment of the polyneuropathy caused by hereditary transthyretin-mediated amyloidosis in adults. It was FDA approved in October 2018. Inotersen has been shown to improve the course of neurologic disease and quality of life in patients with hereditary transthyretin amyloidosis .
Hereditary transthyretin amyloidosis is caused by single-nucleotide variants in the gene encoding transthyretin (TTR), which lead to transthyretin misfolding and the deposition of amyloid substance systemically. Progressive amyloid accumulation may lead to multiorgan dysfunction and death .
- Indication
Inotersen is a transthyretin-directed antisense oligonucleotide indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
- Associated Conditions
- Hereditary Transthyretin Amyloidosis, Polyneuropathies caused by Hereditary transthyretin-mediated amyloidosis
- Associated Therapies
- -
TEGSEDI Pregnancy Surveillance Program
- Conditions
- Interventions
- First Posted Date
- 2020-02-17
- Last Posted Date
- 2024-04-12
- Lead Sponsor
- Akcea Therapeutics
- Target Recruit Count
- 20
- Registration Number
- NCT04270058
- Locations
- 🇺🇸
TEGSEDI Pregnancy Coordinating Center, Morgantown, West Virginia, United States
NEURO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Hereditary Transthyretin-Mediated Amyloid Polyneuropathy
- Conditions
- Interventions
- First Posted Date
- 2019-10-23
- Last Posted Date
- 2024-12-13
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 168
- Registration Number
- NCT04136184
- Locations
- 🇺🇸
Mayo Clinic - Arizona, Scottsdale, Arizona, United States
🇺🇸Mayo Clinic - Jacksonville, Jacksonville, Florida, United States
🇺🇸Indiana University School of Medicine - Indianapolis, Indianapolis, Indiana, United States
24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients
- First Posted Date
- 2018-10-11
- Last Posted Date
- 2020-12-07
- Lead Sponsor
- Brigham and Women's Hospital
- Target Recruit Count
- 50
- Registration Number
- NCT03702829
- Locations
- 🇺🇸
Brigham and Women's Hospital, Boston, Massachusetts, United States
ATTR Expanded Access Program (EAP) by Ionis
- Conditions
- First Posted Date
- 2018-01-17
- Last Posted Date
- 2019-08-05
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Registration Number
- NCT03400098
Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)
- Conditions
- Interventions
- First Posted Date
- 2014-06-26
- Last Posted Date
- 2023-11-18
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 135
- Registration Number
- NCT02175004
- Locations
- 🇺🇸
Indiana University School of Medicine, Indianapolis, Indiana, United States
🇺🇸Johns Hopkins University Bayview Medical Center, Baltimore, Maryland, United States
🇧🇷AACD, Sao Paulo, Brazil
Efficacy and Safety of Inotersen in Familial Amyloid Polyneuropathy
- Conditions
- Interventions
- First Posted Date
- 2012-11-29
- Last Posted Date
- 2019-07-17
- Lead Sponsor
- Ionis Pharmaceuticals, Inc.
- Target Recruit Count
- 173
- Registration Number
- NCT01737398
- Locations
- 🇵🇹
CHP-HGSA, Unidade Clinica de Paramiloidose, Porto, Portugal
🇩🇪UKM; Universitätsklinikum Münster, Klinik für Transplantationsmedizin, Munster, Germany
🇪🇸Hospital Universitari Vall D' Hebron, Barcelona, Spain
Drug Development Updates
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