Vyondys 53
(Golodirsen)Dosage & Administration
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Vyondys 53 Prescribing Information
Dosage and Administration (Application of a topical anesthetic cream to the infusion site prior to administration of VYONDYS 53 may be considered. VYONDYS 53 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion. Infuse the diluted VYONDYS 53 over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medications with VYONDYS 53 or infuse other medications concomitantly via the same intravenous access line with VYONDYS 53. If a hypersensitivity reaction occurs, consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy[see Contraindications , Warnings and Precautions ] . | 06/2024 |
Contraindications (VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53 [see Warnings and Precautions ] .VYONDYS 53 is contraindicated in patients with serious hypersensitivity to golodirsen or to any of the inactive ingredients in VYONDYS 53. | 06/2024 |
Warnings and Precautions (Hypersensitivity reactions, including anaphylaxis, rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in VYONDYS 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy and monitor until the condition resolves [see Dosage and Administration ]. VYONDYS 53 is contraindicated in patients with a history of a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53[see Contraindications ]. | 06/2024 |
VYONDYS 53 is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the
The effect of VYONDYS 53 on dystrophin production was evaluated in one study in DMD patients with a confirmed mutation of the
Study 1 Part 1 was a double-blind, placebo-controlled, dose-titration study in 12 DMD patients. Patients were randomized 2:1 to receive VYONDYS 53 or matching placebo. VYONDYS 53-treated patients received four escalating dose levels, ranging from 4 mg/kg/week (less than the recommended dosage) to 30 mg/kg/week, by intravenous infusion for 2 weeks at each dose level.
Study 1 Part 2 was a 168-week, open-label study assessing the efficacy and safety of VYONDYS 53 at a dose of 30 mg/kg/week in the 12 patients enrolled in Part 1, plus 13 additional treatment-naive patients with DMD amenable to exon 53 skipping. At study entry (either in Part 1 or Part 2), patients had a median age of 8 years and were on a stable dose of corticosteroids for at least 6 months. Efficacy was assessed based on change from baseline in the dystrophin protein level (measured as % of the dystrophin level in healthy subjects, i.e., % of normal) at Week 48 of Part 2. Muscle biopsies were obtained at baseline prior to treatment and at Week 48 of Part 2 in all VYONDYS 53-treated patients (n=25), and were analyzed for dystrophin protein level by Sarepta western blot. Mean dystrophin levels increased from 0.10% (SD 0.07) of normal at baseline to 1.02% (SD 1.03) of normal by Week 48 of Study 1 Part 2, with a mean change in dystrophin of 0.92% (SD 1.01) of normal levels (p<0.001); the median change from baseline was 0.88%.
Individual patient dystrophin levels from Study 1 are shown in Table 2.
Patient Number | Sarepta Western Blot % Normal Dystrophin | Patient number | Sarepta Western Blot % Normal Dystrophin | ||||
| Baseline | Part 2 Week 48 | Change from baseline | Baseline | Part 2 Week 48 | Change from baseline | ||
| 1 | 0.08 | 0.09 | 0.01 | 14 | 0.22 | 0.28 | 0.06 |
| 2 | 0.11 | 0.11 | 0.01 | 15 | 0.14 | 0.21 | 0.07 |
| 3 | 0.21 | 0.22 | 0.01 | 16 | 0.05 | 0.42 | 0.37 |
| 4 | 0.05 | 0.12 | 0.08 | 17 | 0.07 | 1.03 | 0.97 |
| 5 | 0.03 | 0.12 | 0.09 | 18 | 0.02 | 1.57 | 1.55 |
| 6 | 0.06 | 0.14 | 0.09 | 19 | 0.12 | 1.17 | 1.05 |
| 7 | 0.12 | 0.37 | 0.25 | 20 | 0.03 | 1.72 | 1.69 |
| 8 | 0.11 | 1.06 | 0.95 | 21 | 0.11 | 1.77 | 1.66 |
| 9 | 0.06 | 0.54 | 0.48 | 22 | 0.31 | 4.30 | 3.99 |
| 10 | 0.05 | 0.97 | 0.92 | 23 | 0.11 | 0.36 | 0.25 |
| 11 | 0.06 | 1.55 | 1.49 | 24 | 0.03 | 0.91 | 0.88 |
| 12 | 0.07 | 1.91 | 1.84 | 25 | 0.07 | 1.29 | 1.22 |
| 13 | 0.10 | 3.25 | 3.15 | ||||
- Serum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53 ()
2.1 Monitoring to Assess SafetySerum cystatin C, urine dipstick, and urine protein-to-creatinine ratio should be measured before starting VYONDYS 53. Consider measurement of glomerular filtration rate prior to initiation of VYONDYS 53. Monitoring for kidney toxicity during treatment is recommended. Obtain the urine samples prior to infusion of VYONDYS 53 or at least 48 hours after the most recent infusion
[see Warnings and Precautions ]. - 30 milligrams per kilogram once weekly ()
2.2 Dosing InformationThe recommended dosage of VYONDYS 53 is 30 milligrams per kilogram administered once weekly as a 35 to 60-minute intravenous infusion via an in-line 0.2 micron filter.
If a dose of VYONDYS 53 is missed, it may be administered as soon as possible after the scheduled dose.
- Administer as an intravenous infusion over 35 to 60 minutes via an in-line 0.2 micron filter (,
2.2 Dosing InformationThe recommended dosage of VYONDYS 53 is 30 milligrams per kilogram administered once weekly as a 35 to 60-minute intravenous infusion via an in-line 0.2 micron filter.
If a dose of VYONDYS 53 is missed, it may be administered as soon as possible after the scheduled dose.
)2.4 Administration InstructionsApplication of a topical anesthetic cream to the infusion site prior to administration of VYONDYS 53 may be considered.
VYONDYS 53 is administered via intravenous infusion. Flush the intravenous access line with 0.9% Sodium Chloride Injection, USP, prior to and after infusion.
Infuse the diluted VYONDYS 53 over 35 to 60 minutes via an in-line 0.2 micron filter. Do not mix other medications with VYONDYS 53 or infuse other medications concomitantly via the same intravenous access line with VYONDYS 53.
If a hypersensitivity reaction occurs, consider slowing the infusion, interrupting, or discontinuing the VYONDYS 53 therapy[see Contraindications , Warnings and Precautions ]. - Dilution required prior to administration ()
2.3 Preparation InstructionsVYONDYS 53 is supplied in single-dose vials as a preservative-free concentrated solution that requires dilution prior to administration. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. Use aseptic technique.
- Calculate the total dose of VYONDYS 53 to be administered based on the patient's weight and the recommended dose of 30 milligrams per kilogram. Determine the volume of VYONDYS 53 needed and the correct number of vials to supply the full calculated dose.
- Allow the vials to warm to room temperature. Mix the contents of each vial by gently inverting 2 or 3 times. Do not shake.
- Visually inspect each vial of VYONDYS 53. The solution is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles. Do not use if the solution in the vials is cloudy, discolored or contains extraneous particulate matter other than trace amounts of small, white to off-white amorphous particles.
- With a syringe fitted with a 21-gauge or smaller bore non-coring needle, withdraw the calculated volume of VYONDYS 53 from the appropriate number of vials.
- Dilute the withdrawn VYONDYS 53 in 0.9% Sodium Chloride Injection, USP, to make a total volume of 100 to150 mL. Gently invert 2 to 3 times to mix. Do not shake. Visually inspect the diluted solution. Do not use if the solution is cloudy, discolored or contains extraneous particulate matter other than trace amounts of small, white to off-white amorphous particles.
- Administer the diluted solution via an in-line 0.2 micron filter.
- VYONDYS 53 contains no preservatives and should be administered immediately after dilution. Complete infusion of diluted VYONDYS 53 within 4 hours of dilution. If immediate use is not possible, the diluted product may be stored for up to 24 hours at 2°C to 8°C (36°F to 46°F). Do not freeze. Discard unused VYONDYS 53.
VYONDYS 53 is a clear to slightly opalescent, colorless liquid, and may contain trace amounts of small, white to off-white amorphous particles, and available as:
- Injection: 100 mg/2 mL (50 mg/mL) solution in a single-dose vial
There are no human or animal data available to assess the use of VYONDYS 53 during pregnancy. In the U.S. general population, major birth defects occur in 2 to 4% and miscarriage occurs in 15 to 20% of clinically recognized pregnancies.
VYONDYS 53 is contraindicated in patients with a serious hypersensitivity reaction to golodirsen or to any of the inactive ingredients in VYONDYS 53. Anaphylaxis has occurred in patients receiving VYONDYS 53