Instructions for Use Video | Once-weekly SKYTROFA® (lonapegsomatropin-tcgd)

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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:

Acute critical illness after open heart surgery, abdominal surgery or multiple accidental trauma, or those with acute respiratory failure due to risk of increased mortality with use of pharmacologic doses of somatropin

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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:

Acute critical illness after open heart surgery, abdominal surgery or multiple accidental trauma, or those with acute respiratory failure due to risk of increased mortality with use of pharmacologic doses of somatropin
Hypersensitivity to somatropin or any of the excipients in SKYTROFA. Systemic hypersensitivity reactions have been reported with post-marketing use of somatropin products
Closed epiphyses
Active malignancy
Active proliferative or severe non-proliferative diabetic retinopathy
Prader-Willi syndrome who are severely obese, have a history of upper airway obstruction or sleep apnea or have severe respiratory impairment due to the risk of sudden death

WARNINGS AND PRECAUTIONS

Increased mortality in patients with acute critical illness due to complications following open heart surgery, abdominal surgery or multiple accidental trauma, or those with acute respiratory failure has been reported after treatment with pharmacologic doses of somatropin. The safety of continuing SKYTROFA treatment in patients receiving replacement doses for approved indications who concurrently develop these illnesses has not been established
Serious systemic hypersensitivity reactions including anaphylaxis and angioedema have been reported with post-marketing use of somatropin products. Inform patients and caregivers that such reactions are possible and that prompt medical attention should be sought if an allergic reaction occurs. Do not use SKYTROFA in patients with known hypersensitivity to SKYTROFA or any of its excipients
Patients with active malignancy have an increased risk of progression with somatropin treatment. Any preexisting malignancy should be inactive and treatment complete before instituting therapy with SKYTROFA. In childhood cancer survivors treated with radiation to the brain/head for their first neoplasm who developed subsequent GHD treated with somatropin, an increased risk for a second neoplasm has been reported. Monitor patients with a history of GHD secondary to an intracranial neoplasm routinely for progression/recurrence of the tumor. Monitor patients carefully for development of neoplasms and/or increased growth/potential malignant changes of preexisting nevi
Treatment with somatropin may decrease insulin sensitivity, and new onset type 2 diabetes mellitus has been reported. Previously undiagnosed impaired glucose tolerance and overt diabetes mellitus may be unmasked. Patients with type 1 and 2 diabetes mellitus or impaired glucose tolerance should be monitored closely, and antihyperglycemic drugs may require adjustment
Intracranial hypertension (IH) with papilledema, visual changes, headache, nausea, and/or vomiting has been reported in a small number of patients treated with somatropin. Symptoms resolved rapidly after cessation of therapy/reduction of the dose. Perform fundoscopic examination routinely before initiating treatment with SKYTROFA to exclude preexisting papilledema, and periodically thereafter. If papilledema is observed, stop the treatment. If somatropin-induced IH is diagnosed, restart treatment at a lower dose after IH-associated signs and symptoms have resolved
Fluid retention during somatropin therapy may occur. Clinical manifestations of fluid retention (eg, edema, arthralgia, myalgia, nerve compression syndromes including carpal tunnel syndrome/paresthesia) are usually transient and dose dependent
Patients receiving somatropin therapy may be at risk for reduced serum cortisol levels and/or unmasking of central hypoadrenalism. Patients treated with glucocorticoid replacement for previously diagnosed hypoadrenalism may require an increase in their maintenance/stress doses following initiation of SKYTROFA therapy. Monitor patients with known hypoadrenalism for reduced serum cortisol levels and/or need for glucocorticoid dose increases
Undiagnosed or untreated hypothyroidism may prevent response to SKYTROFA. Central hypothyroidism may first become evident or worsen with treatment. Perform periodic thyroid function tests and initiate/properly adjust thyroid hormone replacement therapy when indicated
Slipped capital femoral epiphysis may occur more frequently in patients undergoing rapid growth. Evaluate pediatric patients with the onset of a limp or complaints of persistent hip or knee pain, as slipped capital femoral epiphysis has been reported during lonapegsomatropin treatment. Patients and caregivers should be made aware that osteonecrosis is considered a potential risk for human growth hormone products
Monitor patients for progression of preexisting scoliosis
Cases of pancreatitis have been reported in pediatric patients receiving somatropin. Consider pancreatitis in patients with persistent severe abdominal pain
Lipoatrophy may result when somatropin is administered at the same site over a long period of time. Rotate injection sites to reduce this risk
There have been reports of fatalities after initiating therapy with somatropin in pediatric patients with Prader-Willi syndrome who had one or more of the following risk factors: severe obesity, history of upper airway obstruction or sleep apnea, or unidentified respiratory infection. Male patients with one or more of these factors may be at greater risk than females. SKYTROFA is not indicated for the treatment of pediatric patients who have growth failure due to genetically confirmed Prader-Willi syndrome
Serum levels of inorganic phosphorus, alkaline phosphatase, and parathyroid hormone may increase after somatropin treatment. Monitor abnormal laboratory tests as appropriate

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

Glucocorticoids: SKYTROFA may reduce serum cortisol concentrations, which may require an increase in the dose of glucocorticoids
Cytochrome P450-metabolized drugs: Somatropin may increase cytochrome P450 (CYP450)-mediated antipyrine clearance. Carefully monitor patients using drugs metabolized by CYP450 liver enzymes in combination with SKYTROFA
Oral estrogen: May reduce the response to SKYTROFA. Higher doses of SKYTROFA may be required
Insulin and/or other hypoglycemic agents: SKYTROFA may decrease insulin sensitivity. Patients with diabetes mellitus may require adjustment of insulin or hypoglycemic agents

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

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. Patients aged 6 months to 3 years could be treatment naïve and were included only in the safety and tolerability analyses.³

SKYTROFA was studied in 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. Long-term safety was the primary endpoint.⁴

Patient baseline demographics²*

	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

* Plus-minus values are means ± SD.

^† Genotropin^®.

^‡ Historical growth rates based on best available medical records (SKYTROFA [n = 94] and daily somatropin^† [n = 54]).

A broad range of patients enrolled in the 26-week clinical trial¹

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)

Patient baseline characteristics²

	Full 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

* Baseline indicates last nonmissing measurement before the first dose of lonapegsomatropin-tcgd.²

^† Patients completed SKYTROFA^® treatment (mean treatment duration 3.2 years) because the physician deemed that treatment for pediatric GHD was no longer necessary.²

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Patient baseline demographics2*

A broad range of patients enrolled in the 26-week clinical trial1

Patient baseline characteristics2

Watch: How to use the SKYTROFA^® Auto-Injector

Patient baseline demographics²*

A broad range of patients enrolled in the 26-week clinical trial¹

Patient baseline characteristics²