Danicopan |
Formulary
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Danicopan is recommended, as an add-on to ravulizumab or eculizumab as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults who have residual haemolytic anaemia, only if:
• they have clinically significant extravascular haemolysis while on treatment with a complement component 5 inhibitor (C5 inhibitor) and
• the company provides it according to the commercial arrangement. |
NICE TA1010: Danicopan with ravulizumab or eculizumab for treating paroxysmal nocturnal haemoglobinuria |
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Iptacopan |
Formulary
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Iptacopan is recommended, within its anticipated marketing authorisation, as an option for treating paroxysmal nocturnal haemoglobinuria (PNH) in adults with haemolytic anaemia.
Iptacopan will be available via the Innovative Medicines Fund (IMF) according to a set of treatment criteria which translates the NICE recommendation into a clinical guide as to use in practice. These treatment criteria can be found on the application form(s) on the Blueteq site.
NHS England will then routinely commission iptacopan in patients with PNH via commissioned centres. |
NICE TA1000: Iptacopan for treating paroxysmal nocturnal haemoglobinuria |
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Ravulizumab Ultomiris® |
Formulary
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Concentrate for solution for infusion 300mg/3mL, 1,100mg/11mL, 300mg/30mL Tertiary centres only |
NICE TA698: Ravulizumab for treating paroxysmal nocturnal haemoglobinuria NICE TA710: Ravulizumab for treating atypical haemolytic uraemic syndrome |
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