Source: European Medicines Agency (EU) Revision Year: 2026 Publisher: Akcea Therapeutics Ireland Ltd, St. James House, 72 Adelaide Road, Dublin 2, D02 Y017, Ireland
Tegsedi is indicated for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR).
Treatment should be initiated by and remain under the supervision of a physician experienced in the treatment of patients with hereditary transthyretin amyloidosis.
The recommended dose is 284 mg inotersen by subcutaneous injection. Doses should be administered once every week. For consistency of dosing, patients should be instructed to receive the injection on the same day every week.
Inotersen is associated with reductions in platelet count, which may result in thrombocytopenia. Dosing should be adjusted according to laboratory values as follows:
Table 1. Inotersen monitoring and dosing recommendations according to platelet count:
| Platelet count (x 109/L) | Monitoring frequency | Dosing |
| >100 | Every 2 weeks | Weekly dosing should be continued. |
| ≥75 to <100* | Every week | Dosing frequency should be reduced to 284 mg every 2 weeks. |
| <75* | Twice weekly until 3 successive values above 75 then weekly monitoring. | Dosing should be paused until 3 successive values >100. On reinitiation of treatment dose frequency should be reduced to 284 mg every 2 weeks. |
| <50‡† | Twice weekly until 3 successive values above 75 then weekly monitoring. More frequent monitoring should be considered if additional risk factors for bleeding are present. | Dosing should be paused until 3 successive values >100. On reinitiation of treatment dose frequency should be reduced to 284 mg every 2 weeks. Corticosteroids should be considered if additional risk factors for bleeding are present. |
| <25† | Daily until 2 successive values above 25. Then twice weekly monitoring until 3 successive values above 75. Then weekly monitoring until stable. | Treatment should be discontinued. Corticosteroids recommended. |
* If the subsequent test confirms the initial test result, then monitoring frequency and dosing should be adjusted as recommended in the table.
‡ Additional risk factors for bleeding include age >60 years, receiving anticoagulant or antiplatelet medicinal products, and/or prior history of major bleeding events.
† It is strongly recommended that, unless corticosteroids are contraindicated, the patient receives glucocorticoid therapy to reverse the platelet decline. Patients who discontinue therapy with inotersen due to platelet counts below 25 x 109/L should not reinitiate therapy.
If a dose of inotersen is missed, then the next dose should be administered as soon as possible, unless the next scheduled dose is within two days, in which case the missed dose should be skipped and the next dose administered as scheduled.
No dose adjustment is required in patients aged 65 years and over (see section 5.2).
No dose adjustment is required for patients with mild or moderate renal impairment (see section 5.2). Inotersen should not be used in patients with a urine protein to creatinine ratio (UPCR) ≥113 mg/mmol (1 g/g) or estimated glomerular filtration rate (eGFR) <45 ml/min/1.73 m² (see section 4.3).
Because of the risk of glomerulonephritis and possible renal function decline, UPCR and eGFR should be monitored during treatment with inotersen (see section 4.4). If acute glomerulonephritis is confirmed, permanent discontinuation of the treatment should be considered.
No dose adjustment is required for patients with mild or moderate hepatic impairment (see section 5.2). Inotersen must not be used in patients with severe hepatic impairment (see section 4.3).
Inotersen has not been evaluated in patients undergoing liver transplant. It is, therefore, recommended that inotersen should be discontinued in subjects undergoing liver transplantation.
The safety and efficacy of inotersen in children and adolescents below 18 years of age have not been established. No data are available.
Subcutaneous use only. Each pre-filled syringe is for one-time use only.
The first injection administered by the patient or caregiver should be performed under the guidance of an appropriately qualified health care professional. Patients and/or caregivers should be trained in the subcutaneous administration of Tegsedi.
Sites for injection include the abdomen, upper thigh region, or outer area of the upper arm. It is important to rotate sites for injection. If injected in the upper arm, the injection should be administered by another person. Injection should be avoided at the waistline and other sites where pressure or rubbing from clothing may occur. Tegsedi should not be injected into areas of skin disease or injury. Tattoos and scars should also be avoided.
The pre-filled syringe should be allowed to reach room temperature prior to injection. It should be removed from refrigerated storage at least 30 minutes before use. Other warming methods should not be used.
In the event of an overdose, supportive medical care should be provided including consulting with a healthcare professional and close observation of the clinical status of the patient.
Platelet and renal function tests should be monitored regularly.
5 years.
Tegsedi may be stored unrefrigerated for up to 6 weeks below 30°C. If not used within 6 weeks, it should be discarded.
Store in a refrigerator (2°C–8°C).
Do not freeze.
Store in the original package in order to protect from light.
1.5 mL solution in a clear Type 1 glass pre-filled syringe.
Tray with tear-off lid.
Pack sizes of 1 or 4 pre-filled syringes. Not all pack sizes may be marketed.
Tegsedi should be inspected visually prior to administration. The solution should be clear and colourless to pale yellow. If the solution is cloudy or contains visible particulate matter, the contents must not be injected.
Each pre-filled syringe should be used only once and then placed in a sharps disposal container for disposal.
Any unused medicinal product or waste material should be disposed of in accordance with local requirements.
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