Although we will not be issuing a
formal press release, we wanted you to know that today (Wednesday
4 September 2024) we have recommended iptacopan, a new treatment
option for adults with paroxysmal nocturnal haemoglobinuria with
haemolytic anaemia (PNH).
Around 220 people could benefit from
iptacopan, a pill taken at home twice daily, which is more
convenient than standard care for this condition. Standard care
includes eculizumab and ravulizumab – both given in hospital as
infusions through a line in the vein. If following treatment
people still experience anaemia then pegcetacoplan is a further
treatment option. This is given as
an infusion
into the muscle through the
skin.
Paroxysmal nocturnal
haemoglobinuria (PNH) is
a rare blood condition which causes
persistent anaemia because
the body's immune system attacks the
red blood cells.
The new treatment
helps prevent
anaemia by reducing
the destruction of red blood cells
in the
blood vessels (intravascular
haemolysis) and in the liver and spleen (extravascular
haemolysis).
Evidence from clinical trials shows
that it increases the level of haemoglobin in the blood, reducing
the need for transfusions, and the independent committee found it
to be cost effective for routine treatment on the
NHS.
This is the
1000th
treatment that NICE has
evaluated since April 1999 when it was
formed.
Of these 1000 technology appraisals
undertaken by the independent committees 84% have been positive
recommendations. This has benefitted millions of
babies, children
and adults in England through
providing the best care while ensuring value for money
to the taxpayer.
Iptacopan, (also known as
Fabhalta, and
made by Novartis)
was awarded
its licence from
the Medicines and Healthcare products Regulatory Agency
on 2 August
2024.
-
The final guidance Overview | Iptacopan for
treating paroxysmal nocturnal haemoglobinuria | Guidance |
NICE
-
Section 7 of the National Institute for Health and Care
Excellence (Constitution and Functions) and the Health and
Social Care Information Centre (Functions) Regulations
2013 requires integrated
care boards, NHS England and, with respect to their public
health functions, local authorities to
comply with
the recommendations in this
evaluation within 3 months of its date of
publication.
-
Section f of The Innovative Medicines Fund Principles
states that
a discretionary source of early funding (from the
overall Innovative Medicines
Fund budget)
is available
for certain medicines
recommended by
NICE.
In this instance, interim funding
has been agreed for iptacopan.
Interim funding will end 90 days
after positive final guidance is published, at which point
funding will switch to routine commissioning
budgets.
-
Medicines and Healthcare products Regulatory Agency -
GOV.UK (www.gov.uk)
