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Efficacy and Safety in Treatment-naïve Patients

heiGHt study design

heiGHt was a randomized, open-label, active-controlled, phase 3 study that compared the efficacy, safety, and tolerability of once-weekly SKYTROFA® versus once-daily somatropin in children with GHD1,2*

heiGHt chart
*Annualized height velocity (AHV) at week 52 was the primary endpoint.2
Study Design Details Demographics

Higher mean AHV versus daily somatropin

SKYTROFA demonstrated higher AHV in a phase 3 clinical trial of 161 treatment-naïve pediatric patients with GHD1,2

Mean AHV at week 52: once-weekly SKYTROFA versus daily somatropin1,2†‡

The change in mean bone age from baseline to week 52 was similar with SKYTROFA and daily somatropin,‡ and the observed mean BMI SDS remained within normal range‡ for both groups2,3

†At week 52, patients receiving once-weekly SKYTROFA had an AHV of 11.2 cm/year compared with 10.3 cm/year for those receiving a daily somatropin.1,2 ‡Within ± 2.0 SD of the mean.2

Sustained growth was achieved with SKYTROFA in treatment-naïve patients2

Patients treated with SKYTROFA had a greater increase from baseline in height SDS§ compared with patients treated with a daily somatropin at week 52 (1.10 versus 0.96, respectively)2||

§LS mean by ANCOVA.2 ||Patients treated with SKYTROFA and daily somatropin had an increase in height SDS from baseline (SE) of 1.10 (0.04) and 0.96 (0.05) at week 52, respectively. Estimated difference = 0.14 SDS.2

Established safety profile

The safety profile of SKYTROFA has been established in pediatric patients aged 1 year and older who weigh at least 11.5 kg1

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Low immunogenicity, with no neutralizing antibodies detected2

Only 6.7% (7 of 105) of patients showed detectable binding antibodies through week 52 of the heiGHt trial2

  • Some patients taking SKYTROFA experienced mild injection-site reactions2
  • Systemic hypersensitivity reactions have been reported with postmarketing use of somatropin products1

AEs reported in ≥ 5% of SKYTROFA-treated pediatric patients and more frequently than in daily somatropin-treated# pediatric patients (52 weeks of treatment)1

AEs SKYTROFA
(n = 105)		 Daily somatropin
(n = 56)
n (%) n (%)
Viral infection 16 (15%) 6 (11%)
Pyrexia 16 (15%) 5 (9%)
Cough 11 (11%) 4 (7%)
Nausea and vomiting 11 (11%) 4 (7%)
Hemorrhage 7 (7%) 1 (2%)
Diarrhea 6 (6%) 3 (5%)
Abdominal pain 6 (6%) 2 (4%)
Athralgia and arthritis# 6 (6%) 1 (2%)
The etiology of hemorrhage in the SKYTROFA treatment group included epistaxis (n = 3), contusion (n = 2), petechiae (n = 1), and eye hemorrhage (n = 1).1 #The etiology of arthralgia and arthritis in the SKYTROFA treatment group included arthralgia (n = 5) and reactive arthritis (n = 1).1
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IMPORTANT SAFETY INFORMATION

INDICATIONS AND USAGE

SKYTROFA® (lonapegsomatropin-tcgd) injection is a human growth hormone (GH) indicated for the:

  • Treatment of pediatric patients aged 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous GH
  • Replacement of endogenous GH in adults with growth hormone deficiency (GHD)

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 somatropin
  • Hypersensitivity to somatropin or any of the excipients in SKYTROFA

Click here to contact Ascendis Pharma Medical Information or call 1-844-442-7236.

IMPORTANT SAFETY INFORMATION

INDICATIONS AND USAGE

SKYTROFA® (lonapegsomatropin-tcgd) injection is a human growth hormone (GH) indicated for the:

  • Treatment of pediatric patients aged 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous GH
  • Replacement of endogenous GH in adults with growth hormone deficiency (GHD)

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 somatropin
  • Hypersensitivity to somatropin or any of the excipients in SKYTROFA
  • Pediatric patients with closed epiphyses
  • Active malignancy
  • Active proliferative or severe non-proliferative diabetic retinopathy
  • Pediatric patients with 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: Increased mortality has been reported after treatment with somatropin in patients with acute critical illness due to complications following open-heart surgery, abdominal surgery, multiple accidental trauma, and in patients with acute respiratory failure
  • Severe Hypersensitivity: Serious systemic hypersensitivity reactions including anaphylaxis and angioedema have been reported with post-marketing use of somatropin products, including SKYTROFA. Inform patients and/or caregivers that such reactions are possible and that prompt medical attention should be sought if an allergic reaction occurs
  • Increased Risk of Neoplasms: There is an increased risk of malignancy progression with somatropin treatment in patients with active malignancy. Any preexisting malignancy should be inactive, and its treatment complete prior to instituting SKYTROFA. In childhood cancer survivors treated with radiation to the brain/head for their first neoplasm who developed subsequent GHD and were treated with somatropin, an increased risk of a second neoplasm has been reported. Children with certain rare genetic causes of short stature have an increased risk of developing malignancies and should be carefully monitored for development of neoplasms. Monitor patients with a history of GHD secondary to an intracranial neoplasm for progression/recurrence of the tumor. Monitor patients carefully for development of neoplasms and/or increased growth/potential malignant changes of preexisting nevi. Advise patients/caregivers to report changes in the appearance of preexisting nevi
  • Glucose Intolerance and Diabetes Mellitus: Treatment with somatropin may decrease insulin sensitivity, particularly at higher doses. Previously undiagnosed impaired glucose tolerance and overt type 2 diabetes mellitus may be unmasked. Monitor glucose levels in all patients, especially those with risk factors for type 2 diabetes mellitus, such as obesity or a family history of type 2 diabetes mellitus. When initiating SKYTROFA, monitor patients with preexisting type 1 or type 2 diabetes mellitus or impaired glucose tolerance closely, and adjust the doses of antihyperglycemic drugs as needed
  • Intracranial Hypertension: 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 usually occurred within 8 weeks of the initiation of somatropin and resolved rapidly after cessation of therapy/reduction of the dose. Perform fundoscopic examination prior to initiation of treatment and periodically thereafter. If papilledema is observed, stop the treatment. If somatropin-induced IH is confirmed, restart SKYTROFA treatment at a lower dose after IH-associated signs and symptoms have resolved
  • Fluid Retention: May occur during somatropin therapy. Clinical manifestations of fluid retention (eg, edema, arthralgia, myalgia, nerve compression syndromes including carpal tunnel syndrome/paresthesia) are usually transient and dose dependent
  • Hypoadrenalism: Patients receiving somatropin therapy who have or are at risk for pituitary hormone deficiency(s) may be at risk for reduced serum cortisol levels and/or unmasking of central (secondary) 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
  • Hypothyroidism: Undiagnosed/untreated hypothyroidism may prevent an optimal response to SKYTROFA. Monitor thyroid function periodically as hypothyroidism may occur or worsen after initiation of SKYTROFA
  • Slipped Capital Femoral Epiphysis in Pediatric Patients: Slipped capital femoral epiphysis may occur more frequently in patients undergoing rapid growth and may lead to osteonecrosis. Evaluate pediatric patients receiving SKYTROFA with the onset of a limp or complaints of persistent hip or knee pain for slipped capital femoral epiphysis and osteonecrosis, and manage accordingly
  • Progression of Preexisting Scoliosis in Pediatric Patients: Monitor patients with a history of scoliosis for disease progression
  • Pancreatitis: Cases of pancreatitis have been reported in pediatric patients receiving somatropin. The risk may be greater in pediatric patients than in adults. Consider pancreatitis in patients with persistent severe abdominal pain
  • Lipoatrophy: Lipoatrophy may result when somatropin is administered at the same site over a long period of time. Rotate injection sites to reduce this risk
  • Sudden Death in Pediatric Patients With Prader-Willi Syndrome: 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 female patients. SKYTROFA is not indicated for the treatment of pediatric patients who have growth failure due to genetically confirmed Prader-Willi syndrome
  • Laboratory Tests: Serum levels of alkaline phosphatase and phosphate may increase after SKYTROFA therapy. Serum levels of parathyroid hormone may increase after somatropin treatment. If a patient is found to have abnormal laboratory tests, monitor as appropriate

ADVERSE REACTIONS

  • Pediatric patients with GHD: the most common adverse reactions (≥ 5%) in patients treated with SKYTROFA and more frequently than in those treated with daily somatropin were viral infection, pyrexia, cough, nausea and vomiting, hemorrhage, diarrhea, abdominal pain, and arthralgia and arthritis
  • Adult patients with GHD: the most common adverse reaction (≥ 5%) in patients treated with SKYTROFA and more frequently than in those treated with placebo were edema and central (secondary) hypothyroidism

DRUG INTERACTIONS

  • Glucocorticoids: Patients treated with glucocorticoid replacement for hypoadrenalism may require an increase in their maintenance or stress doses following initiation of SKYTROFA
  • Pharmacologic Glucocorticoid Therapy and Supraphysiologic Glucocorticoid Treatment: Adjust glucocorticoid dosing in pediatric patients to avoid both hypoadrenalism and an inhibitory effect on growth
  • Cytochrome P450-Metabolized Drugs: SKYTROFA may alter the clearance. Monitor carefully if used with SKYTROFA
  • Oral Estrogen: Patients receiving oral estrogen replacement may require higher SKYTROFA dosages
  • Insulin and/or Other Antihyperglycemic Agents: Dose adjustment of insulin and/or antihyperglycemic agent may be required for patients with diabetes mellitus

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 SKYTROFA full Prescribing Information.

References: 1. SKYTROFA. Prescribing information. Ascendis Pharma, Inc.; 2024 2. Thornton PS, Maniatis AK, Aghajanova E, et al. Weekly lonapegsomatropin in treatment-naïve children with growth hormone deficiency: the phase 3 heiGHt trial. J Clin Endocrinol Metab. 2021;106(11):3184-3195. doi:10.1210/clinem/dgab529 3. Ascendis Pharma, Inc. Data on file; 2020

ANCOVA = analysis of covariance; BMI = body mass index; CI = confidence interval; GH = growth hormone; GHD = growth hormone deficiency; hGH = human growth hormone; IGF-1 = insulin-like growth factor-1; LS = least squares; SD = standard deviation; SDS = standard deviation score; SE = standard error.