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HomeAbout NGENLAEfficacy & SafetyPhase 3 Main Study + SafetyPhase 3 OLE Study + SafetyPhase 2 Study + SafetyDosingDosingDosingAdministrationMyNgenla PlanIGF-1 MonitoringAccess & SupportAccess &
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Prescribing InformationIndication Patient Site
Once-weekly NGENLAUp to 8 years of clinical data1‑3

Phase 3 Main Study + Safety

Phase 3 OLE Study + Safety

Phase 2 Study + Safety

Phase 3 study designEvaluated vs daily GENOTROPIN for injection in a phase 3 study of 224 patients with pediatric growth hormone deficiency (pGHD)

Mean height velocity in phase 3 main study1,2,4

Efficacy and safety of once-weekly NGENLA were assessed in a phase 3 open-label, multicenter, randomized, active-controlled, noninferiority, parallel-group study vs daily GENOTROPIN in treatment-naïve, prepubertal children with pGHD. After a 12-week screening period, patients were randomized 1:1 to weekly injections of NGENLA 0.66 mg/kg/week (n=109) or daily GENOTROPIN 0.034 mg/kg/day (n=115) for 12 months.1,2

12-month, phase 3, noninferiority study end points 

PRIMARY END POINT1

  • Annual height velocity in centimeters/year after 12 months of treatment

KEY SECONDARY END POINTS2

  • Change in height standard deviation score (SDS) at 6 and 12 months, compared to baseline
  • Absolute insulin-like growth factor 1 (IGF-1) and IGF-1 SDS levels after dosing with NGENLA
0.034 mg/kg/day is the equivalent of 0.24 mg/kg/week.Mean annualized height velocity1NGENLA has efficacy comparable to GENOTROPIN® (somatropin)1 CI, confidence interval.Mean increase in height standard deviation score (SDS) Modeled SDS insulin-like growth factor (IGF-1) normalization over 1 year with NGENLA2,4 Three patients had a mean IGF-1 SDS of ≥2; no patients had IGF-1 SDS ≥3.3.4IGF-1 was to be measured on day 4 (96 hours) post dose, as this interval has been shown to provide the best estimate of the mean IGF-1 value over the dosing interval of 1 week in this post hoc analysis. As patients had samples drawn at variable days post dose, a model was developed to calculate estimated mean IGF-1 SDS values from sampling over the dosing interval (1 week), regardless of the timing of IGF-1 measurement since last dose.4These post hoc results should be interpreted with caution because the analyses were not prespecified and therefore potentially subject to a greater degree of bias.4Phase 3 safety results1

Adverse reactions reported in ≥5% of patients taking NGENLA at 1 year

Adverse event GENOTROPIN®
(n=115) n (%)		 NGENLA
(n=109) n (%)
Injection site reactions* 29 (25.2) 46 (42.2)
Nasopharyngitis† 33 (28.7) 36 (33)
Headache 25 (21.7) 18 (16.5)
Pyrexia 17 (14.8) 18 (16.5)
Anemia 10 (8.7) 10 (9.2)
Cough 9 (7.8) 9 (8.3)
Vomiting 9 (7.8) 8 (7.3)
Hypothyroidism 3 (2.6) 7 (6.4)
Adverse event GENOTROPIN®
(n=115) n (%)		 NGENLA
(n=109) n (%)
Injection site reactions* 29 (25.2) 46 (42.2)
Nasopharyngitis† 33 (28.7) 36 (33)
Headache 25 (21.7) 18 (16.5)
Pyrexia 17 (14.8) 18 (16.5)
Anemia 10 (8.7) 10 (9.2)
Cough 9 (7.8) 9 (8.3)
Vomiting 9 (7.8) 8 (7.3)
Hypothyroidism 3 (2.6) 7 (6.4)
  • Only 1 NGENLA patient discontinued due to an AE‡
Injection site reactions included injection site pain (39% NGENLA vs 25% daily GENOTROPIN), injection site swelling/induration/hypertrophy/inflammation (10% NGENLA vs 1% daily GENOTROPIN), injection site erythema (8% NGENLA vs none daily GENOTROPIN), injection site pruritus (5% NGENLA vs none daily GENOTROPIN), injection site hemorrhage (5% NGENLA vs none daily GENOTROPIN).1Nasopharyngitis included rhinitis, pharyngitis, rhinitis allergic, pharyngitis streptococcal, viral pharyngitis, nasopharyngitis.1Discontinuation due to injection site erythema and induration.OLE, open-label extension.References:NGENLA. Prescribing information. Pfizer Inc.; 2023.Data on file. Pfizer Inc., New York, NY.Zadik Z, Zelinska N, Iotova V, et al. Long-term efficacy and safety of once-weekly somatrogon in pediatric subjects with growth hormone deficiency: results from up to 8 years of somatrogon treatment. Poster presented at: Annual Meeting of the Endocrine Society 2023; June 15-18, 2023.Deal CL. Results of global phase 3 study of once-weekly somatrogon in growth hormone deficient children. Poster presented at: Annual Meeting of the Endocrine Society 2020; June 8-22, 2020.
Phase 3 open-label extension (OLE) study design2,4Evaluated vs daily GENOTROPIN® (somatropin) for injection in a phase 3 study of 224 patients with pGHD2,4
Study design4
  • Open-label, single-arm extension, phase 3 study continuation of the randomized 12-month study 
Dosing2
  • Patients who received NGENLA during the phase 3 study continued with the same dose during the OLE 
  • Patients who received GENOTROPIN during the phase 3 study were switched to NGENLA and began treatment with a dose of 0.66 mg/kg/week 
  • The dose of NGENLA can be adjusted every 3 months based on the patient's body weight and may be decreased for safety reasons based on predefined dose-adjustment criteria
End points4†
  • AHV, change in height SDS 
  • Insulin-like growth factor 1 (IGF-1) levels and IGF-1 SDS assessed on day 4 after NGENLA dosing across study visits 
Study participants2
  • 212 patients completed the phase 3 study and enrolled in the OLE 
  • Patients who received NGENLA in the phase 3 study continued to receive NGENLA 0.66 mg/kg/week (n=104) 
  • Patients who received GENOTROPIN in the phase 3 study were switched to NGENLA 0.66 mg/kg/week (n=108)
Key inclusion criteria4
  • Patients who completed the phase 3 study and provided their consent were eligible to be enrolled
0.034 mg/kg/day is the equivalent of 0.24 mg/kg/week.Efficacy and safety were assessed every 3 months for the first year of OLE.Mean AHV for the phase 3 OLE5

Study limitations
The primary objective of the phase 3 OLE was to evaluate the efficacy and safety of NGENLA. Any inference of clinical significance should be interpreted with caution as the study was open label and lacked a comparator arm.5

These efficacy data represent the pivotal study year (year 1), as well as the first 3 years of the phase 3 OLE (year 2, year 3, and year 4). Statistical analyses of these data are descriptive, and no formal hypothesis testing was performed.5

Patients with HV data as of March 2022. Error bars represent SD.5Mean height standard deviation score (SDS) at month 12 of each study year4

Study limitations
The primary objective of the phase 3 OLE was to evaluate the efficacy and safety of NGENLA. Any inference of clinical significance should be interpreted with caution as the study was open label and lacked a comparator arm.4

These efficacy data represent the pivotal study year (year 1), as well as the first 3 years of the phase 3 OLE (year 2, year 3, and year 4). Statistical analyses of these data are descriptive, and no formal hypothesis testing was performed.4

Patients with HV data as of March 2022. Error bars represent SD.4Change in mean height SDS (from baseline) at month 12 of each study year4

Study limitations
The primary objective of the phase 3 OLE was to evaluate the efficacy and safety of NGENLA. Any inference of clinical significance should be interpreted with caution as the study was open label and lacked a comparator arm.4

These efficacy data represent the pivotal study year (year 1), as well as the first 3 years of the phase 3 OLE (year 2, year 3, and year 4). Statistical analyses of these data are descriptive, and no formal hypothesis testing was performed.4

Patients with HV data as of March 2022. Error bars represent SD.4Phase 3 OLE safety results4

A phase 3 open-label extension continues to monitor patients’ AEs

SUMMARY OF ALL-CAUSALITY TEAEs (MAIN STUDY AND OLE)4

Study limitations
The primary objective of the phase 3 OLE was to evaluate the efficacy and safety of NGENLA. Any inference of clinical significance should be interpreted with caution as the study was open label and lacked a comparator arm. Statistical analyses of these data are descriptive, and no formal hypothesis testing was performed.4

AE, adverse event; TEAE, treatment-emergent adverse event.References:NGENLA. Prescribing information. Pfizer Inc.; 2023.Data on file. Pfizer Inc., New York, NY.Zadik Z, Zelinska N, Iotova V, et al. Long-term efficacy and safety of once-weekly somatrogon in pediatric subjects with growth hormone deficiency: results from up to 8 years of somatrogon treatment. Poster presented at: Annual Meeting of the Endocrine Society 2023; June 15-18, 2023.Wajnrajch M, Miller BS, Steelman J, et al. Switch data from the open-label extension of the pivotal phase 3 study of once weekly somatrogon compared with daily somatropin in pediatric patients with growth hormone deficiency (GHD). Poster presented at: Annual Meeting of the Endocrine Society 2021; March 20-23, 2021. Poster 7129.Steelman J, Choe J, Stawerska R, et al. Open-label extension of a global phase 3 study of once-weekly somatrogon in pediatric patients with growth hormone deficiency: results from up to 4 years of somatrogon treatment. Poster presented at: 2024 Pediatric Endocrine Society Annual Meeting; May 2-5, 2024. Poster 21.
Phase 2 safety and dose-finding study design2 Phase 2 randomized, open-label, dose-finding study comprised of 5 treatment periods up to 8 years. In the main study (Periods I and II), 53 prepubertal children with GHD were randomized to once-weekly NGENLA (0.25, 0.48, or 0.66 mg/kg/week) or once-daily GENOTROPIN (0.034 mg/kg/day); 48 continued into the OLE, consisting of Period III (original NGENLA dose, GENOTROPIN recipients randomized to one of three NGENLA doses), Period IV (NGENLA 0.66 mg/kg/week), and Period V (prefilled NGENLA pen [0.66 mg/kg/week]).2,3

End points2
Primary end point
  • Safety* 
Secondary end points
  • AHV 
  • Change in height SDS 
  • Change in IGF-1 SDS 
Inclusion criteria4
  • Prepubertal children aged ≥3 years and <11 years (girls), <12 years (boys) 
  • Isolated GHD or GHD due to multiple pituitary hormone deficiency (on stable replacement therapy for ≥3 months) 
  • Peak plasma GH level ≤10 ng/mL on 2 different provocative tests (insulin tolerance/arginine/clonidine/glucagon, with or without propranolol/L‑dopa plus propranolol) 
  • Bone age ≤ chronological age <10 years (girls), <11 years (boys) 
  • Impaired height (≥SD below the mean); impaired AHV SDS < -0.7 (≤25th percentile for chronological age) 
  • BMI within +2 SD of mean BMI 
  • Baseline IGF-1 SDS ≤ -1.0 
  • Normal karyotype for girls
  • No signs or symptoms of intracranial hypertension
AHV, annualized height velocity; BMI, body mass index; GH, growth hormone; GHD growth hormone deficiency; IGF‑1, insulin-like growth factor 1; SD, standard deviation; SDS, standard deviation score.All patients randomized to receive 1 of the 3 NGENLA doses started treatment for 2 weeks with the lowest NGENLA dose, which based on the patient's dose allocation, was followed by a dose increase to the next dose level every 2 weeks until the final allocated dose was reached.5Safety evaluations included vital signs, injection site reactions, immunogenic response, and laboratory safety assessments. The safety monitoring for most safety evaluations was initially done weekly and then monthly in the first 6 months, then every 3 months until end of study.2Phase 2 open-label extension (OLE) study design2

Phase 2 OLE study design2†
In extension year 1 (n=48), patients taking NGENLA continued their assigned dose. Patients taking GENOTROPIN® (somatropin) were re-randomized to 1 of the 3 NGENLA doses. A total of 14 patients continued on the FDA-approved 0.66 mg/kg/week dose from the main year. 
In extension years 2 and beyond, patients were transitioned to NGENLA 0.66 mg/kg/week.

End points2
Primary end point

  • Safety

Secondary end points

  • AHV 
  • Change in height SDS 
  • Annual bone maturation
Key inclusion criteria2
Patients who completed 12 months of treatment in the safety and dose-finding portion of the main study. 

Study participants2
  • 48 of 53 patients who completed the main study were randomized and entered the OLE study 
  • At the start of year 1 of OLE, the majority (66.7%) of patients were male and almost all (93.8%) were White 
AHV, annualized height velocity; FDA, Food and Drug Administration; SDS, standard deviation score.The number of patients reflects the new patient numbers for each arm after GENOTROPIN re-randomization.2Most patients switched from the single-use vials to a multidose pen after the completion of extension year 3. Extension year 4 is the first year where patients were evaluated on the prefilled pen device.2Year 2, n=44; year 3, n=43; and year 4, n=38.2NGENLA has up to 8 years of height standard deviation score (SDS) data2,3,5
Study limitations
  • The primary objective of the phase 2 OLE was to evaluate safety of NGENLA. Any inference of clinical significance should be interpreted with caution as the study was open label and lacked a comparator arm. Statistical analyses of these data are descriptive, and no formal hypothesis testing was performed2* 
  • Results for efficacy outcomes were unable to be measured in year 5 as patients were switched from vial and syringe to the prefilled pen device on a rolling basis. Efficacy outcomes measurement resumes in year 6 with a complete patient population switched to the prefilled pen device3
Phase 2 randomized, open-label, dose-finding study comprised of 5 treatment periods up to 8 years. In the main study (Periods I and II), 53 prepubertal children with GHD were randomized to once-weekly NGENLA (0.25, 0.48, or 0.66 mg/kg/week) or once-daily GENOTROPIN (0.034 mg/kg/day); 48 continued into the OLE, consisting of Period III (original NGENLA dose, GENOTROPIN recipients randomized to one of three NGENLA doses), Period IV (NGENLA 0.66 mg/kg/week), and Period V (prefilled NGENLA pen [0.66 mg/kg/week]).4Phase 2 safety data3The safety profile of NGENLA was evaluated across 8 years in a phase 2 open-label extension

THE OVERALL INCIDENCE OF TEAEs IN PATIENTS DURING THE OLE3 TEAE, treatment-emergent adverse event.References:NGENLA. Prescribing information. Pfizer Inc.; 2023.Data on file. Pfizer Inc., New York, NY. Zadik Z, Zelinska N, Iotova V, et al. Long-term efficacy and safety of once-weekly somatrogon in pediatric subjects with growth hormone deficiency: results from up to 8 years of somatrogon treatment. Poster presented at: Annual Meeting of the Endocrine Society 2023; June 15-18, 2023.Zelinska N, Iotova V, Skorodok J, et al. Long-acting C-terminal peptide–modified hGH (MOD-4023): results of a safety and dose-finding study in GHD children. J Clin Endocrinol Metab. 2017;102(5):1578-1587. doi:10.1210/jc.2016-3547Zadik Z, Zelinska N, Iotova V, et al. An open-label extension of a phase 2 dose-finding study of once-weekly somatrogon vs. once-daily Genotropin in children with short stature due to growth hormone deficiency: results following 5 years of treatment. J Pediatr Endocrinol Metab. 2023;36(3):261-269. doi:10.1515/jpem-2022-0359
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INDICATIONNGENLA is indicated for the treatment of pediatric patients aged 3 years and older who have growth failure due to an inadequate secretion of endogenous growth hormone.
IMPORTANT SAFETY INFORMATIONCONTRAINDICATIONS
Somatrogon 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 the risk of increased mortality with growth hormones.NGENLA is contraindicated in patients with hypersensitivity to somatrogon or any of its excipients. Somatrogon is contraindicated in patients with closed epiphyses. Somatrogon is contraindicated in patients with active malignancy due to the risk of malignancy progression. Somatrogon is contraindicated in patients with active proliferative or severe non-proliferative diabetic retinopathy. Somatrogon is contraindicated in 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 due to complications following open heart surgery, abdominal surgery, or multiple accidental trauma, or those with acute respiratory failure has been reported with somatropin. The safety of continuing NGENLA treatment for the approved indication who concurrently develop these illnesses has not been established.Severe systemic hypersensitivity reactions including anaphylactic reactions and angioedema have been reported with somatropin. Inform patients and caregivers that such reactions are possible and that prompt medical attention should be sought if allergic reaction occurs.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 should be completed, prior to instituting therapy with NGENLA. Discontinue NGENLA if there is evidence of recurrent malignancy. In childhood cancer survivors who were treated with radiation to the brain/head for their first neoplasm and who developed subsequent GHD and were treated with somatropin, an increased risk of a second neoplasm has been reported. Intracranial tumors, in particular meningiomas, were the most common of these second neoplasms. Monitor all patients with a history of GHD secondary to an intracranial neoplasm while on NGENLA therapy for progression or recurrence of the tumor. Because children with certain rare genetic causes of short stature have an increased risk of developing malignancies, thoroughly consider the risks and benefits of starting NGENLA in these patients. If treatment with NGENLA is initiated, carefully monitor these patients for development of neoplasms. Monitor patients on NGENLA therapy carefully for increased growth or potential malignant changes of preexisting nevi. Advise patients and/or caregivers to report marked changes in behavior, onset of headaches, vision disturbances, and/or changes in skin pigmentation or changes in the appearance of preexisting nevi. Treatment with growth hormone may decrease insulin sensitivity, particularly at higher doses. New-onset type 2 diabetes mellitus has been reported in patients receiving growth hormone. Patients with undiagnosed pre-diabetes and diabetes mellitus may experience worsened glycemic control and become symptomatic. Monitor glucose levels periodically in all patients receiving NGENLA, especially in those with risk factors for diabetes mellitus, such as obesity, Turner syndrome, or a family history of diabetes mellitus. Patients with preexisting type 1 or type 2 diabetes mellitus or pre-diabetes should be monitored closely. The doses of antidiabetic agents may require adjustment when NGENLA is initiated. Intracranial hypertension (IH) with papilledema, visual changes, headache, nausea, and/or vomiting has been reported in patients treated with somatropin. Symptoms usually occurred within the first 8 weeks after the initiation of somatropin therapy. In all reported cases, IH-associated signs and symptoms rapidly resolved after cessation of therapy or a reduction of somatropin dose. NGENLA should be temporarily discontinued in patients with clinical or fundoscopic evidence of IH. If IH is confirmed, restart treatment with NGENLA at a lower dose after IH-associated signs and symptoms have resolved. Fluid retention during NGENLA therapy may occur. Clinical manifestations of fluid retention (eg, edema and nerve compression syndromes including carpal tunnel syndrome/paresthesia) are usually transient and dose dependent. Patients receiving growth hormone therapy who have or are at risk for pituitary hormone deficiencies may be at risk for reduced serum cortisol levels and/or unmasking of central (secondary) hypoadrenalism. In addition, patients treated with glucocorticoid replacement for previously diagnosed hypoadrenalism may require an increase in their maintenance or stress doses following initiation of NGENLA treatment. Monitor patients for reduced serum cortisol levels and/or need for glucocorticoid dose increases in those with known hypoadrenalism. Undiagnosed/untreated hypothyroidism may prevent an optimal response to NGENLA therapy. In patients with GH deficiency, central (secondary) hypothyroidism may first become evident or worsen during treatment with growth hormone therapy. Therefore, patients should have periodic thyroid function tests and thyroid hormone replacement therapy should be initiated or appropriately adjusted 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. NGENLA increases growth rate, and progression of preexisting scoliosis can occur in patients who experience rapid growth. Growth hormone treatment has not been shown to increase the occurrence of scoliosis. Monitor patients with a history of scoliosis for disease progression. Cases of pancreatitis have been reported in patients receiving somatropin. The risk may be greater in pediatric patients compared with adults. Consider pancreatitis in patients who develop persistent severe abdominal pain. When growth hormone is administered subcutaneously at the same site over a long period of time, lipoatrophy may result. Rotate injection sites when administering NGENLA to reduce this risk. There have been reports of sudden death 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. NGENLA is not indicated for the treatment of pediatric patients who have growth failure due to genetically confirmed Prader-Willi syndrome. Serum levels of phosphorus, alkaline phosphatase, and parathyroid hormone may increase with NGENLA therapy. If a patient is found to have abnormal laboratory tests, monitor as appropriate. ADVERSE EFFECTS
Adverse reactions reported in ≥5% of patients treated with NGENLA are injection site reactions, nasopharyngitis, headache, pyrexia, anemia, cough, vomiting, hypothyroidism, abdominal pain, rash, and oropharyngeal pain. Health care providers should supervise the first injection and provide appropriate training and instruction for the proper use of all NGENLA devices.
INDICATION
NGENLA is indicated for the treatment of pediatric patients aged 3 years and older who have growth failure due to an inadequate secretion of endogenous growth hormone.