Non-acquired isolated growth hormone deficiency Advocacy Hub | UniteRare

At a Glance

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Regulatory Watchboard

2 events

Jan 2026Assessment of Body Composition in Children Treated With Growth Hormone for the Indication of Isolated Non-acquired Growth Hormone Deficiency.

Assistance Publique - Hôpitaux de Paris

TrialRECRUITING

Jun 2023

Ngenla: FDA approved

treatment of pediatric patients aged 3 years and older who have growth failure due to an inadequate secretion of endogenous growth hormone

FDAcompleted

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

3 programs

FINANCIAL LANDSCAPE SUMMARY

Total programs

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Patient Assistance Programs3

Ngenla

Pfizer Ireland Pharmaceuticals

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Skytrofa

Ascendis Pharma Endocrinology Division A/S

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Macrelin

Aeterna Zentaris GmbH

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Approved Treatments

3 FDA-approved

Skytrofa

(lonapegsomatropin-tcgd)Orphan drug

Ascendis Pharma Endocrinology Division A/S

Recombinant Human Growth Hormone [EPC]

12.1 Mechanism of Action SKYTROFA is a pegylated human growth hormone (somatropin) for once-weekly subcutaneous injection [see Clinical Pharmacology (...

Approved Jul 2025FDA label ↗

Ngenla

(somatrogon-ghla)Orphan drug

Pfizer Ireland Pharmaceuticals

12.1 Mechanism of Action Somatrogon-ghla binds to the GH receptor and initiates a signal transduction cascade culminating in changes in growth and met...

Approved Jun 2023FDA label ↗

Macrelin

(macimorelin acetate)Orphan drugstandard

Aeterna Zentaris GmbH

Growth Hormone Secretagogue Receptor Agonist [EPC]

12.1 Mechanism of Action Macimorelin stimulates GH release by activating growth hormone secretagogue receptors present in the pituitary and hypothalam...

FDA label ↗

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