Children with growth disturbance could be offered a new weekly treatment as an alternative to daily injections.
The National Institute for Health and Care Excellence (NICE) has recommended somatrogon (Ngenla, Pfizer) after an appraisal committee found it was as effective as somatropin (Genotropin, Pfizer, and biosimilars), which is currently recommended as treatment.
Somatrogon has a marketing authorisation for treating growth disturbance caused by growth hormone deficiency in children and young people aged 3 years and over.
Somatrogon is a glycoprotein comprised of the amino acid sequence of human growth hormone (hGH) with one copy of the of C-terminal peptide (CTP) from the beta chain of human chorionic gonadotropin (hCG) at the N-terminus and two copies of CTP (in tandem) at the C-terminus. Once administered by subcutaneous injection, it binds to the growth hormone receptor and initiates a signal transduction cascade, culminating in changes in growth and metabolism.
A Less Painful Alternative
After reviewing evidence from clinical trials, NICE decided in final draft guidance that somatrogon was as effective as one preparation of somatropin, and suggested in a press release that children treated with somatrogon "could be spared the pain and discomfort caused by daily injections". It estimated that around 2200 children could benefit from the guidance.
Helen Knight, director of medicines evaluation at NICE, described the recommendation as "a welcome development".
The cost of treatment with somatrogon at the recommended dose of 0.66 mg per kilogram per week is estimated at £9,500 a year, based on a patient weighing 40 kg. The guidance recommended that clinicians should assess which treatments were suitable for their patient and choose the least expensive following discussions about their advantages and disadvantages.
Light Touch Procedure
Somatrogon is the first medicine to be recommended by NICE to have gone through all its steps so far under the new 'light touch' proportionate approach to technology appraisals procedure. As a result, the evaluation was completed 7 weeks faster than the previous cost-comparison process of 29 weeks by simplifying, removing, or reconfiguring some of the usual steps.
NICE expected to "improve this even further in future topics under this new proportionate way of working", Ms Knight said. She added: "We want to get the best care to patients fast, while ensuring value for money for the taxpayer, and at the same time creating useful and useable advice for the NHS.
"NICE is already one of the quickest health technology assessment bodies in the world to appraise new medicines. This pilot will help us maintain a flexible and proportionate approach to appraisals and further improve the speed with which we provide recommendations on promising new treatments."
The guidance applies to England only, although implementation of advice is usually followed by the NHS in Wales. Final guidance for somatrogon is expected to be published by NICE in February 2023.