SomatrogonNgenla
Treatment of growth hormone deficiency in paediatric patients, specifically for short stature associated with biochemical growth hormone deficiency (SSABGHD) and short stature and slow growth (SSSG) in patients who do not have a mature skeleton.
Decision on record
- Meeting Mar 2022 Recommended Paediatric growth hormone deficiency
Access path
- TGA registered · Ngenla
TGA label narrower than the PBS population
- Mar 2022Recommended · restricted
vs somatropin
- PBS listing · Authority Required
From the public summary
The PBAC recommended the Section 100 Growth Hormone Program listing of somatrogon for the treatment of SSABGHD and SSSG in patients who do not have a mature skeleton (i.e. a bone age of less than 13.5 years in females or less than 15.5 years in males).PSD · Mar 2022
The PBAC noted the consumer comments which noted the improved patient satisfaction and treatment convenience of weekly dosing with somatrogon compared to daily dosing with somatropin. The PBAC agreed with the consumer comments that there is clinical need for a once-weekly growth hormone treatment for patients with paediatric GHD.PSD · Mar 2022
The submission presented a cost-minimisation analysis of once-weekly somatrogon compared to daily somatropin in paediatric patients for the treatment of SSABGHD, and SSSG. The submission assumed non-inferior effectiveness to somatropin in terms of annual HV at 12 months, and non-inferior safety in terms of AEs at 12 months. The use of a cost-minimisation was appropriate considering that the claim of non- inferiority was mostly supported by the evidence. 13PSD · Mar 2022
Somatrogon was assumed to be non-inferior to somatropin for the treatment of pGHD Therapeutic claim: safety with respect to AEs at 12 months.PSD · Mar 2022
The submission described somatrogon as non-inferior in terms of effectiveness and safety when compared to somatropin. This claim was mostly supported by the evidence presented in the submission. The key issues included:PSD · Mar 2022
• The clinical trial evidence was that of pre-pubertal children (Study 4004: <11 years for girls and <12 for boys; Studies 4006 and 4009: <10 years for girls and <11 years for boys) whereas the proposed PBS restrictions are for patients with a bone age (up to 13.5 for girls and 15.5 years for boys).PSD · Mar 2022
The PBAC noted and welcomed the input from individuals (12), health care professionals (2) and organisations (1) via the Consumer Comments facility on the PBS website. The comments from carers described the benefits of a once-weekly GHD treatment, including improved compliance, increased flexibility, fewer injection site effects and reduced damage to skin, and an overall reduced disease burden and improved quality of life.PSD · Mar 2022
The PBAC noted the advice received from the Australian Pituitary Foundation (APF) clarifying the likely use of somatrogon in clinical practice. The PBAC specifically noted the advice that the use of somatrogon may reduce physical irritation and damage at the injection site, reduce the emotional and psychological burden that both children and their parents/carers experience with daily injections, and minimise the impact on family dynamics.PSD · Mar 2022
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated. PBAC assessment based on cost-effectiveness being acceptable if cost-minimised to somatropin.
Decision context
PopulationPaediatric patients with growth hormone deficiency, specifically those with short stature associated with biochemical growth hormone deficiency or short stature and slow growth, with a bone age less than 13.5 years in females or less than 15.5 years in males, aged 3 years or older.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2022 | Recommended · restricted | somatropin | — | RCT · Non-inferior effectiveness and safety (annualised height velocity, change in height SDS, IGF-1 and IGFBP-3 levels) |
Consumer voice
Consumer input from individuals, healthcare professionals, and the Australian Pituitary Foundation highlighted benefits of once-weekly GHD treatment including improved compliance, reduced injection burden, fewer injection site effects, and improved quality of life—particularly enabling children to participate in normal childhood activities and reducing emotional/psychological burden on patients an
The comments from carers described the benefits of a once-weekly GHD treatment, including improved compliance, increased flexibility, fewer injection site effects and reduced damage to skin, and an overall reduced disease burden and improved quality of life. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with biochemical GHD confirmation, specific stature criteria, and skeletal maturity thresholds absent from TGA label.