UK – Roche’s injectable C5 inhibitor PiaSky recommended by NICE for rare blood disorder PNH

Roche’s injectable component C5 inhibitor has been recommended by the National Institute for Health and Care Excellence (NICE) to treat the rare blood disorder paroxysmal nocturnal haemoglobinuria (PNH).

PiaSky (crovalimab), which can be self-administered by patients following adequate training, will be available on the NHS in England and Wales as a monotherapy for eligible adults and adolescents aged 12 years and older and weighing at least 40kg.

Affecting approximately 1.59 per every 100,000 people in the UK, PNH occurs when the red blood cells are destroyed by the complement system. This results in symptoms such as anaemia, fatigue, blood clots, and can lead to kidney disease.

Current C5 inhibitors, including AstraZeneca’s Soliris (eculizumab) and Ultomiris (ravulizumab), block part of the complement system cascade and can be effective in treating the condition, but require regular intravenous infusions…