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inotersen (Tegsedi)
Indications:
- treatment of hereditary transthyretin amyloidosis
- treatment of associated polyneuropathy [3] dosage:
- 300 mg sq weekly
Adverse effects:
- glomerulonephritis (3%)*
- thrombocytopenia (3%)*
- mortality (4%)
* black box warning
Laboratory:
- platelet count
- renal function test
- urinalysis with reflex urine microscopy
Mechanism of action:
- inhibits hepatic synthesis of transthyretin
General
short interfering double-stranded RNA; antisense oligonucleotide (siRNA, microRNA, miRNA)
metabolic agent (metabolic modifier)
References
- Benson MD, Waddington-Cruz M, Berk JL et al
Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
N Engl J Med 2018; 379:22-31. July 5.
PMID: 29972757
https://www.nejm.org/doi/full/10.1056/NEJMoa1716793
- Yasgur BS
New Agents Show Promise for Severe and Fatal Genetic Disease.
Medscape - Jul 10, 2018.
https://www.medscape.com/viewarticle/899112
- George J
FDA Greenlights Inotersen for Rare Disease Nerve Pain -
But approval comes with boxed warning and mandatory monitoring.
MedPage Today, October 06, 2018
https://www.medpagetoday.com/neurology/generalneurology/75546
- RxNorm
- Brooks M
FDA OKs Inotersen (Tegsedi) for Hereditary ATTR With Polyneuropathy.
Medscape - Oct 08, 2018.
https://www.medscape.com/viewarticle/903104