China-based Changchun GeneScience Pharmaceutical Co., Ltd announced that it has received another indication approval from the National Medical Products Administration (NMPA) for its somatropin to treat Prader-Willi Syndrome (PWS). This latest approval marks the tenth indication for the company’s somatropin and represents the first approval for a home-grown growth hormone to treat PWS.
Drug Profile and PWS Background
PWS, also known as hypotonia-mental retardation-hypogonadal development-obesity syndrome, is a genetic disorder caused by defects in genomic imprinting, with an incidence rate between 1/10,000 and 1/30,000. Between 40-100% of PWS patients experience short stature due to growth hormone deficiency. Recombinant human growth hormone treatment has been shown to provide multiple benefits for PWS patients, including improvements in height, lean body mass, exercise capacity, behavior, and cognition. In the US, recombinant human growth hormone was approved to treat PWS-related short stature in children in 2000.
Previous Approvals
GeneScience’s somatropin has previously been approved for use in the following indications:
- Slow growth in children due to endogenous growth hormone deficiency
- Short stature in children due to Noonan syndrome
- Short stature or growth failure in children due to SHOX gene deficiency
- Short stature in children due to achondroplasia
- Growth failure in girls due to gonadal dysgenesis (Turner syndrome)
- Short bowel syndrome in adults receiving nutritional support
- Growth hormone deficiency due to established hypothalamic-pituitary disease symptoms and significant growth hormone deficiency confirmed by two different growth hormone stimulation tests
- Severe burns and short stature due to small for gestational age (SGA)-Fineline Info & Tech
