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.
PiaSky works in a similar way, but can be given as a subcutaneous injection every four weeks, following one intravenous loading dose and four additional subcutaneous weekly loading doses in the first month of treatment.
NICE’s final draft guidance was supported by positive results from the late-stage COMMODORE 2 trial, which demonstrated that PiaSky was non-inferior to Soliris in the co-primary endpoints of haemolysis control and transfusion avoidance.
The drug will initially be made available through NHS England’s Innovative Medicines Fund, which facilitates faster access to new treatments while further data is collected.
Alexa Urspruch, UK lead for solid tumours and rare indications, Roche Products Limited, said: “We are delighted with [this] news that eligible patients in England and Wales will be able to access PiaSky on the NHS… We are working with the PNH National Service to incorporate PiaSky into the patient pathway and we remain committed to ensuring that this treatment is available across the rest of the UK as quickly as possible.”
The recommendation comes just one month after PiaSky was approved by the European Commission for PNH. The drug, which was engineered by Chugai Pharmaceutical Co, also holds approvals in other major markets such as the US and Japan.
