Drug updated on 3/28/2024
| Dosage Form | Injection (subcutaneous: 284 mg/1.5 mL) |
| Drug Class | Transthyretin-directed antisense oligonucleotides |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Summary
- Inotersen (Tegsedi) is indicated for the treatment of polyneuropathy in adults with hereditary transthyretin-mediated amyloidosis.
- A total of 5 systematic reviews and meta-analyses were reviewed, providing insights into the effectiveness and safety profile of Inotersen (Tegsedi).
- The drug has shown significant benefit in high-quality randomized controlled trials for TTR-related amyloid polyneuropathy, as highlighted by one review.
- However, another study found that an indirect comparison or network meta-analysis between Inotersen (Tegsedi), tafamidis, and patisiran was not feasible due to differences in trial designs, baseline population characteristics, outcome definitions and baseline risk across their pivotal trials.
- One review noted conflicting results on cardiac imaging parameters with Inotersen (Tegsedi) use while another pointed out a potential increase in mortality rate compared to placebo along with more dropouts due to adverse events during its clinical trial.
- Despite these concerns raised about its safety profile relative to other treatments like tafamidis or patisiran which have also been approved for ATTR-PN patients; it's important that clinicians consider individual patient characteristics when making prescribing decisions given observed cross-trial heterogeneity among these drugs' studies according to one review.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Tegsedi (inotersen) Prescribing Information. | 2022 | Sobi, Inc. Waltham, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Targeted therapies for hereditary peripheral neuropathies: systematic review and steps towards a 'treatabolome'. | 2021 | Journal of Neuromuscular Diseases |
| Targeting transthyretin - Mechanism-based treatment approaches and future perspectives in hereditary amyloidosis. | 2021 | Journal or neurochemistry |
| Clinical review report: inotersen (Tegsedi). | 2020 | CADTH |
| Pharmacological treatment for familial amyloid polyneuropathy. | 2020 | Cochrane Database of Systematic Reviews |
| Specific therapy for transthyretin cardiac amyloidosis: a systematic literature review and evidence-based recommendations. | 2020 | Journal of the American Heart Association |
| The impact of clinical heterogeneity on conducting network meta-analyses in transthyretin amyloidosis with polyneuropathy. | 2020 | Current Medical Research and Opinion |
| Inotersen and patisiran for hereditary transthyretin amyloidosis: effectiveness and value. | 2018 | ICER |
| Assessment report: Tegsedi. | 2018 | EMA |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Diagnosis and screening of patients with hereditary transthyretin amyloidosis (hATTR): current strategies and guidelines. | 2020 | Therapeutics and Clinical Risk Management |