Active Ingredient: Onasemnogene abeparvovec
Onasemnogene abeparvovec is indicated for the treatment of:
For this indication, competent medicine agencies globally authorize below treatments:
Intravenous
16.5 - 115.5 mL
From 16.5 To 115.5 mL once every day
For single-dose intravenous infusion only.
Patients will receive a dose of nominal 1.1 × 1014 vg/kg onasemnogene abeparvovec. The total volume is determined by patient body weight.
Table 1 gives the recommended dosing for patients who weigh 2.6 kg to 21.0 kg.
Table 1. Recommended dosing based on patient body weight:
| Patient weight range (kg) | Dose (vg) | Total volume of dosea (mL) |
|---|---|---|
| 2.6–3.0 | 3.3 × 1014 | 16.5 |
| 3.1–3.5 | 3.9 × 1014 | 19.3 |
| 3.6–4.0 | 4.4 × 1014 | 22.0 |
| 4.1–4.5 | 5.0 × 1014 | 24.8 |
| 4.6–5.0 | 5.5 × 1014 | 27.5 |
| 5.1–5.5 | 6.1 × 1014 | 30.3 |
| 5.6–6.0 | 6.6 × 1014 | 33.0 |
| 6.1–6.5 | 7.2 × 1014 | 35.8 |
| 6.6–7.0 | 7.7 × 1014 | 38.5 |
| 7.1–7.5 | 8.3 × 1014 | 41.3 |
| 7.6–8.0 | 8.8 × 1014 | 44.0 |
| 8.1–8.5 | 9.4 × 1014 | 46.8 |
| 8.6–9.0 | 9.9 × 1014 | 49.5 |
| 9.1–9.5 | 1.05 × 1015 | 52.3 |
| 9.6–10.0 | 1.10 × 1015 | 55.0 |
| 10.1–10.5 | 1.16 × 1015 | 57.8 |
| 10.6–11.0 | 1.21 × 1015 | 60.5 |
| 11.1–11.5 | 1.27 × 1015 | 63.3 |
| 11.6–12.0 | 1.32 × 1015 | 66.0 |
| 12.1–12.5 | 1.38 × 1015 | 68.8 |
| 12.6–13.0 | 1.43 × 1015 | 71.5 |
| 13.1–13.5 | 1.49 × 1015 | 74.3 |
| 13.6–14.0 | 1.54 × 1015 | 77.0 |
| 14.1–14.5 | 1.60 × 1015 | 79.8 |
| 14.6–15.0 | 1.65 × 1015 | 82.5 |
| 15.1–15.5 | 1.71 × 1015 | 85.3 |
| 15.6–16.0 | 1.76 × 1015 | 88.0 |
| 16.1–16.5 | 1.82 × 1015 | 90.8 |
| 16.6–17.0 | 1.87 × 1015 | 93.5 |
| 17.1–17.5 | 1.93 × 1015 | 96.3 |
| 17.6–18.0 | 1.98 × 1015 | 99.0 |
| 18.1–18.5 | 2.04 × 1015 | 101.8 |
| 18.6–19.0 | 2.09 × 1015 | 104.5 |
| 19.1–19.5 | 2.15 × 1015 | 107.3 |
| 19.6–20.0 | 2.20 × 1015 | 110.0 |
| 20.1–20.5 | 2.26 × 1015 | 112.8 |
| 20.6–21.0 | 2.31 × 1015 | 115.5 |
a NOTE: Number of vials per kit and required number of kits is weight-dependent. Dose volume is calculated using the upper limit of the patient weight range.
An immune response to the adeno-associated viral vector serotype 9 (AAV9) capsid will occur after administration of onasemnogene abeparvovec. This can lead to elevations in liver transaminases, elevations of troponin I, or decreased platelet counts. To dampen the immune response immunomodulation with corticosteroids is recommended. Where feasible, the patient’s vaccination schedule should be adjusted to accommodate concomitant corticosteroid administration prior to and following onasemnogene abeparvovec infusion.
Prior to initiation of the immunomodulatory regimen and prior to administration of onasemnogene abeparvovec, the patient must be checked for symptoms of active infectious disease of any nature.
Starting 24 hours prior to infusion of onasemnogene abeparvovec it is recommended to initiate an immunomodulatory regimen following the schedule below (see Table 2). Deviations from these recommendations are at the discretion of the treating physician.
Table 2. Pre- and post-infusion immunomodulatory regimen:
| Pre-infusion | 24 hours prior to onasemnogene abeparvovec | Prednisolone orally 1 mg/kg/day (or equivalent) |
| Post-infusion | 30 days (including the day of administration of onasemnogene abeparvovec) | Prednisolone orally 1 mg/kg/day (or equivalent) |
| Followed by 28 days: For patients with unremarkable findings (normal clinical exam, total bilirubin, and whose ALT and AST values are both below 2 × upper limit of normal (ULN) at the end of the 30 days period: or For patients with liver function abnormalities at the end of the 30 days period: continuing until the AST and ALT values are below 2 × ULN and all other assessments return to normal range, followed by tapering over 28 days | Tapering of prednisolone (or equivalent), e.g. 2 weeks at 0.5 mg/kg/day and then 2 weeks at 0.25 mg/kg/day oral prednisolone Systemic corticosteroids (equivalent to oral prednisolone 1 mg/kg/day) | |
| Liver transaminases should be monitored for at least 3 months following onasemnogene abeparvovec infusion. | ||
Consult expert(s) if patients do not respond adequately to the equivalent of 1 mg/kg/day oral prednisolone.
If another corticosteroid is used by the physician in place of prednisolone, similar considerations and approach to taper the dose after 30 days should be taken as appropriate.
Onasemnogene abeparvovec is administered as a single-dose intravenous infusion. It should be administered with a syringe pump as a single intravenous infusion with a slow infusion of approximately 60 minutes. It must not be administered as an intravenous push or bolus.
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