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Marketing authorisation without orphan designation - Europe

Therapeutic indication


* Infants, children and adolescents
- Growth disturbance due to insufficient secretion of growth hormone (GH),
- Growth disturbance associated with Turner syndrome,
- Growth disturbance (current height standard deviation score (SDS) < -2,5 and parental adjusted SDS < -1) in short children/adolescents borhttp://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-_Summary_for_the_public/human/000607/WC500043689.pdfn small for gestational age (SGA), with a birth weight and/or length below -2 standard deviation (SD), who failed to show catch-up growth (height velocity (HV) SDS < 0 during the last year) by 4 years of age or later,
- Prader-Willi syndrome (PWS), for improvement of growth and body composition. The diagnosis of PWS should be confirmed by appropriate genetic testing.

* Adults
Replacement therapy in adults with pronounced growth hormone deficiency. Patients with severe growth hormone deficiency in adulthood are defined as patients with known hypothalamic pituitary pathology and at least one known deficiency of a pituitary hormone not being prolactin. These patients should undergo a single dynamic test in order to diagnose or exclude a growth hormone deficiency. In patients with childhood onset isolated GH deficiency (no evidence of hypothalamic-pituitary disease or cranial irradiation), two dynamic tests should be recommended, except for those having low IGF-I concentrations (SDS < -2) who may be considered for one test. The cut-off point of the dynamic test should be strict.

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