Europe – Draft NICE decision rejects Respreeza for incurable, genetic disorder

NICE has published draft guidelines rejecting NHS use of CSL Behring’s Respreeza for treating emphysema in adults with severe alpha-1-proteinase inhibitor deficiency.

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 enzymes.

Therefore, people with the condition are at high risk of the chronic lung condition emphysema as a result of infections and environmental toxins, including tobacco smoke and pollution.

Current therapies, such as bronchodilators and inhaled corticosteroids, strive to delay the worsening of emphysema and manage its symptoms but do not target the cause of the condition…