Active Ingredient: Volanesorsen
Volanesorsen is indicated as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.
For this indication, competent medicine agencies globally authorize below treatments:
For:
Subcutaneous, 285 milligrams volanesorsen, once weekly, over the duration of 3 months. Afterwards, subcutaneous, 285 milligrams volanesorsen, once every 2 weeks.
Treatment should be initiated by and remain under the supervision of a physician experienced in the treatment of patients with FCS. Prior to initiating volanesorsen, secondary causes of hypertriglyceridemia (e.g. uncontrolled diabetes, hypothyroidism) should be excluded or appropriately addressed.
The recommended starting dose is 285 mg in 1.5 ml injected subcutaneously once weekly for 3 months. Following 3 months, dose frequency should be reduced to 285 mg every 2 weeks.
However, treatment should be discontinued in patients with a reduction in serum triglycerides <25% or who fail to achieve serum triglycerides below 22.6 mmol/L after 3 months on volanesorsen 285 mg weekly.
After 6 months of treatment with volanesorsen, increase of dose frequency to 285 mg weekly should be considered if response has been inadequate in terms of serum triglyceride reduction as evaluated by the supervising experienced specialist and in the condition that platelet counts are in the normal range. Patients should be re-downtitrated to 285 mg every 2 weeks if the higher 285 mg once weekly dose does not provide significant additional triglyceride reduction after 9 months.
Patients should be instructed to give the injection on the same day of the week, according to medically determined frequency of administration.
If a dose is missed and noticed within 48 hours, the patient should be directed to give the missed dose as soon as possible. If not noticed within 48 hours, then the missed dose should be skipped and the next planned injection given.
Before initiation of treatment, platelet count should be measured. If the platelet count is below 140 × 109/L another measurement should be taken approximately a week later to reassess. If platelet count remains below 140 × 109/L upon a second measurement, volanesorsen should not be initiated.
After commencing treatment, patients should have platelet levels monitored at least every two weeks, depending on the platelet levels.
Treatment and monitoring should be adjusted according to laboratory values in line with Table 1.
For any patient dose paused or discontinued due to severe thrombocytopenia, the benefits and risks of returning to treatment once platelet count ≥100 × 109/L should be carefully considered. For discontinued patients, a haematologist should be consulted prior to resuming treatment.
Table 1. Volanesorsen monitoring and treatment recommendations:
| Platelet Count (x 109/l) | Dose (285 mg prefilled syringe) | Monitoring Frequency |
|---|---|---|
| Normal (≥140) | Starting dose: Weekly After 3 months: Every 2 weeks | Every 2 weeks |
| 100 to 139 | Every 2 weeks | Weekly |
| 75 to 99 | Pause treatment for ≥4 weeks and resume treatment after platelet levels ≥100 × 109/L | Weekly |
| 50 to 74 | Pause treatment for ≥4 weeks and resume treatment after platelet levels ≥100 × 109/L | Every 2-3 days |
| Less than 50a | Discontinue treatment Glucocorticoids recommended | Daily |
a Consultation of a haematologist is needed to reconsider the benefit/risk for possible further treatment with volanesorsen.
No starting dose adjustment is necessary for elderly patients. There is limited clinical data for patients aged 65 and over.
It is important to rotate sites for injection. Sites for injection include the abdomen, upper thigh region, or outer area of the upper arm. 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 may occur from clothing. This medicinal product should not be injected into tattoos, moles, birthmarks, bruises, rashes, or areas where the skin is tender, red, hard, bruised, damaged, burned, or inflamed.
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