Completed Lungs & Breathing Pregnancy, Children & Inherited Conditions

The Development of a Human Growth Hormone Nasal Spray to Treat Paediatric Growth Hormone Deficiency.

Funder Wellcome Charity-funded
Recipient organisation Critical Pharmaceuticals Limited Source-published name: Critical Pharmaceuticals Limited
Funding £1.5M
Period Sept 2009 — Aug 2013

In plain English

AI plain-English summary

Every day, children with growth hormone deficiency must endure a painful injection—but a new nasal spray could replace the needle entirely. This matters because up to 49% of paediatric patients do not stick to their daily injection regimen, and 70% are unhappy with self-injecting. Poor adherence reduces treatment effectiveness and drives up healthcare costs. The current global market for growth hormone therapy was worth $2.8 billion in 2007, yet the only delivery method remains an injection. Critical Pharmaceuticals has developed CriticalSorb, a nasal delivery system that allows growth hormone to be absorbed through the nose, replacing the daily jab with a simple spray. If this Phase 1 clinical trial succeeds, children with growth hormone deficiency could avoid painful daily injections, dramatically improving adherence and treatment outcomes. The impact would be felt directly in paediatric clinics and family homes, where a spray instead of a needle could transform a child’s experience of chronic care. This is not fundamental science—it is a targeted technology transfer project aimed at bringing an existing innovation into clinical testing, with the goal of changing how a standard therapy is delivered.

View original technical description
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

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Researchers

Illum (EPMC Awardee)

Related Research

Grants with similar aims, by meaning.

Diagnosis of Childhood Growth Hormone Deficiency and Prediction of Therapeutic Response Using Gene Expression Data
A trial comparing the effect and safety of once weekly dosing of somapacitan with daily Norditropin® in children with growth hormone deficiency
Understanding adherence to weekly growth hormone treatment: A prospective, mixed methods study
Development of a long-acting growth hormone antagonist to address the unmet need for treatment in acromegaly
The Growth Hormone Deficiency Reversal Trial: Effect on final height of discontinuation vs continuation of growth hormone treatment in pubertal children with isolated growth hormone deficiency – A non-inferiority randomised controlled trial

Original classification

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