See the committee papers for full details of the evidence. Paroxysmal nocturnal haemoglobinuria (PNH) is a rare blood condition caused by an acquired mutation of the PIG‑A gene within bone marrow stem ...
Novartis’ Fabhalta (iptacopan) has been recommended by the National Institute for Health and Care Excellence (NICE) to treat paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic ...
Roche’s PiaSky is the first monthly subcutaneous therapy for PNH approved in the European Union. Credit: Poetra.RH/Shutterstock. The European Commission (EC) has approved Roche’s recycling monoclonal ...