The development of a human growth hormone nasal spray to treat paediatric growth hormone deficiency. (360G-Wellcome-089791_Z_09_A)

Human growth hormone (hGH) therapy is used to treat growth hormone deficiency (GHD) and other paediatric conditions e.g. Turners syndrome, idiopathic short stature and low gestational weight. The market for hGH replacement therapy was $2.8billion in 2007. hGH replacement therapy requires daily injection of hGH. Studies show that 36-49% of paediatric patients do not adhere to prescribed regimens, 70% are unhappy with injecting themselves daily, and 30% are considering stopping their treatment. This reduces efficacy and increases healthcare costs. There is a significant unmet clinical need for treatment options that can increase patient adherence. Critical Pharmaceuticals has developed CriticalSorb an efficient novel nasal hGH delivery system that allows replacement of a daily injection with a daily nasal spray of hGH. This nasal spray will increase patient adherence by avoiding painful and complex administration procedures. A technology transfer award will allow a clinical assessment of the effectiveness of the hGH nasal spray in a Phase 1 clinical trial. The key goals of the proposed research are to: " Optimise the current prototype nasal formulation " Obtain human pharmacokinetic, pharmacodynamic and nasal tolerability data on lead formulations " Obtain long term non-clinical nasal tolerability data on the lead formulation