NICE has rejected CSL Behring’s alpha1-proteinase inhibitor Respreeza for treating genetic emphysema in the draft guidance, saying that it would cost a massive £8 million to give a patient a year of quality life with the treatment. NHS England’s cost-effectiveness watchdog, the National Institute for Health and Care Excellence (NICE), issued a first draft document saying that it did not currently recommend the drug. A1PI deficiency is an incurable, rare and life-limiting genetic disorder in which a lack of the protein A1PI makes the body, especially the lungs, vulnerable to attack from its own infection-fighting enzyme, neutrophil elastase.
The enzyme is released by neutrophils and macrophages to fight infections but if it is uncontrolled, it can damage tissue – particularly the lungs – if there insufficient inhibitor, alpha-1 antitrypsin.