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IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS
SKYTROFA is contraindicated in patients with:
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INDICATION
SKYTROFA® is a human growth hormone (GH) indicated for the treatment of pediatric patients aged ≥ 1 years weighing ≥ 11.5 kg with growth failure due to inadequate secretion of endogenous GH.
IMPORTANT SAFETY
INFORMATION
CONTRAINDICATIONS
SKYTROFA is contraindicated in patients with:
WARNINGS AND PRECAUTIONS
ADVERSE REACTIONS
The most common adverse reactions (≥ 5%) in patients treated with SKYTROFA were: viral infection (15%), pyrexia (15%), cough (11%), nausea and vomiting (11%), hemorrhage (7%), diarrhea (6%), abdominal pain (6%), and arthralgia and arthritis (6%).
DRUG INTERACTIONS
You are encouraged to report side effects to FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Ascendis Pharma at 1-844-442-7236.
Please click here for full Prescribing Information for SKYTROFA.
heiGHt was a 52-week, randomized, open-label, active-controlled, parallel-group phase 3 study of 161 treatment-naïve, prepubertal (Tanner stage I) pediatric patients with GHD aged 3 years and older. Patients were randomized in a 2:1 ratio to receive either SKYTROFA® 0.24 mg/kg/week (n = 105) or daily somatropin using Genotropin® 0.24 mg/kg/week (n = 56). The primary endpoint was AHV at 52 weeks.1,2
https://doi.org/10.1210/clinem/dgab529Key inclusion criteria2
Patients were excluded based on the following2
SKYTROFA® 0.24 mg hGH/kg/week |
Daily somatropin† 0.24 mg hGH/kg/week |
Total | |
---|---|---|---|
(n = 105) | (n = 56) | (N = 161) | |
Male, n (%) | 86 (82) | 46 (82) | 132 (82) |
White, n (%) | 100 (95.2) | 52 (92.9) | 152 (94.4) |
Chronological age (year) | 8.5 ± 2.7 | 8.5 ± 2.8 | 8.5 ± 2.7 |
Bone age (year) | 5.8 ± 2.6 | 6.0 ± 2.7 | 5.9 ± 2.6 |
Bone age/chronological age (ratio) | 0.69 ± 0.16 | 0.70 ± 0.14 | 0.69 ± 0.15 |
Weight (kg) | 21.0 ± 6.5 | 21.2 ± 6.7 | 21.1 ± 6.6 |
BMI (kg/m2) | 16.1 ± 1.8 | 16.5 ± 2.2 | 16.2 ± 1.9 |
Height (cm) | 112.9 ± 14.1 | 112.2 ± 15.3 | 112.7 ± 14.5 |
Height (SDS) | –2.89 ± 0.85 | –3.00 ± 0.90 | 2.93 ± 0.87 |
Historical growth rate (cm/year)‡ | 3.9 ± 2.0 | 3.9 ± 1.7 | 3.9 ± 1.9 |
IGF-1 SDS | –2.08 ± 0.88 | –1.96 ± 0.98 | 2.04 ± 0.92 |
fliGHt was a multicenter, phase 3, open-label, 26-week trial investigating the safety, tolerability, and efficacy of SKYTROFA® administered once weekly in children with GHD. The trial included 3 treatment-naïve and 143 treatment-experienced patients previously treated with daily hGH for ≤ 130 weeks. Previous daily GH treatments included HumatroPen®, Genotropin Pen®, Saizen one.click®, Omnitrope® Pen, Genotropin GoQuick®, Genotropin MiniQuick®, Norditropin FlexPro®, and Nutropin AQ® NuSpin®. The mean daily somatropin dose was 0.29 mg/kg/week upon entering the trial. Safety and tolerability were the primary endpoints.1 Patients aged 6 months to 3 years could be treatment naïve and were included only in the safety and tolerability analyses.
https://doi.org/10.1159/000524003Key inclusion criteria1:
Patients were excluded based on the following1:
Baseline demographics and clinical characteristics | Total enrolled | Previously treated |
---|---|---|
N = 146 | n = 143 | |
Male, n (%) | 110 (75.3) | 109 (76.2) |
White, n (%) | 124 (84.9) | 121 (84.6) |
Hispanic or Latino, n (%) | 10 (6.8) | 10 (7.0) |
Mean chronological age (year) | 10.6 | 10.8 |
BMI SDS, mean (SD) | –0.3 (1.1) | –0.24 (1.06) |
Height SDS, mean (SD) | –1.42 (0.84) | –1.40 (0.83) |
Tanner stage, n (%) | ||
I | 95 (65.1) | 92 (64.3) |
II | 14 (9.6) | 14 (9.8) |
III | 30 (20.5) | 30 (21.0) |
IV | 7 (4.8) | 7 (4.9) |
IGF‑1 SDS, mean (SD) | 0.85 (1.29) | 0.91 (1.25) |
Daily somatropin dose at trial enrollment (mg hGH/kg/week) | n/a | 0.29 (0.05) |
Daily somatropin dose duration since treatment initiation (years) | n/a | 1.14 (0.73) |
enliGHten was an open-label extension study of pediatric patients with GHD who had previously participated in phase 3 SKYTROFA® trials. The study enrolled a total of 298 patients: 103 patients on SKYTROFA and 55 patients on daily somatropin from the heiGHt trial, and 140 patients on SKYTROFA from the fliGHt trial. The mean age at baseline of the enliGHten trial was 9.5 years for SKYTROFA-treated patients from the heiGHt trial, 9.5 years for daily somatropin-treated patients from the heiGHt trial, and 11.1 years for patients from the fliGHt trial. Treatment completion status was evaluated throughout the study, and the results were stratified accordingly. Long-term safety was the primary endpoint.2
https://doi.org/10.1210/clinem/dgac217Full analysis set | Treatment completers* | |
---|---|---|
(N = 298) | (n = 81)† | |
Age, years, mean (SD) | 10.3 (3.4) | 13.3 (1.8) |
Sex, male, n (%) | 235 (78.9) | 66 (81.5) |
Height SDS, mean (SD) | –1.6 (0.9) | –1.6 (0.7) |
IGF‑1 SDS, mean (SD) | 1.0 (1.3) | 0.8 (1.5) |
Tanner stage, n (%) | ||
Stage I | 214 (71.8) | 22 (30.1) |
Stage II | 40 (13.4) | 17 (23.3) |
Stage III | 25 (8.4) | 28 (38.4) |
Stage IV | 16 (5.4) | 6 (8.2) |
Stage V | 3 (1.0) | 0 |