Versartis Long-Acting Growth Hormone in Children Compared to Daily rhGH

This study is designed as a pivotal study to compare the safety and efficacy of a selected
dose regimen of somavaratan to daily rhGH. The study is a randomized, multi-center, open
label study of 12 months duration. The primary endpoint is height velocity at 12 months.

Inclusion Criteria:

- Chronological Age ≥ 3.0 years and ≤ 10.0 (girls) and ≤ 11.0 (boys).

- Pre-pubertal status: Absent breast development in girls, testicular volume < 4.0 mL in
boys.

- Diagnosis of GHD as documented by two or more GH stimulation test results ≤ 10.0
ng/mL.

- Height SD score ≤ -2.0 at screening.

- Weight for Stature ≥ 10th percentile.

- IGF-I SD score ≤ -1.0 at screening.

- Delayed bone age (≥ 6 months).

Exclusion Criteria:

- Prior treatment with any growth promoting agent

- History of or concurrent significant disease (e.g. diabetes, cystic fibrosis, renal
insufficiency).

- Chromosomal aneuploidy, significant gene mutations (other than those that cause GHD)
or confirmed diagnosis of a named syndrome.

- A diagnosis of Attention Deficit Hyperactivity Disorder.

- Daily use of anti-inflammatory doses of glucocorticoid.

- Prior history of leukemia, lymphoma, sarcoma or cancer.

- Treatment with an investigational drug in the 30 days prior to screening.

- Known allergy to constituents of the study drug formulation.

- Ocular findings suggestive of increased intracranial pressure and/or retinopathy at
screening.

- Significant spinal abnormalities including scoliosis, kyphosis and spina bifida
variants.

- Significant abnormality in screening laboratory studies