NICE Enables Access to First Treatment That Targets the Underlying Pathophysiology of Rare Disease X-Linked Hypophosphataemia (XLH) in Adults1
June 21 2024 - 5:13AM
Business Wire
Kyowa Kirin is delighted by the decision made
today by the National Institute for Health and Care Excellence
(NICE) to recommend CRYSVITA® for the treatment of adults living
with X-linked hypophosphataemia (XLH) in England, Wales and
Northern Ireland
Adults living with the rare disease X-linked hypophosphataemia
(XLH) in England, Wales and Northern Ireland will now have access
to the first available treatment to tackle the underlying cause of
XLH following NICE’s decision to recommend burosumab (marketed as
Crysvita) to treat adults who have a confirmed diagnosis of XLH,
following a new access deal.1
This decision ensures equal access to burosumab across the UK,
as adults living with XLH in Scotland have been able to access
burosumab on the NHS since March 2023, following approval by the
Scottish Medicines Consortium (SMC) through their ultra-orphan
pathway.2 Burosumab is also available for eligible children and
adolescents in all four UK nations.
Initially, NICE published an interim negative decision in its
Appraisal Consultation Document (ACD). Following constructive
engagement between Kyowa Kirin, NICE and NHS England, this
recommendation was revised and an agreement for long term
commissioning reached, thus resulting in a positive decision in its
Final Draft Guidance (FDG).
XLH is a very rare genetic condition, affecting roughly 1.7 –
4.8 per 100,000 people.3 This rare metabolic bone condition is a
life-long and progressive disease that typically presents in early
childhood, causing bowed legs, stunted growth, and bone and joint
pain.4 It is a whole life, whole body, and whole family disease,
according to people living with the condition, as several family
members are often impacted due to its inherited nature.5 XLH also
has an adverse impact on emotional wellbeing for various reasons
including pain, uncertainty about the future, and may also be
associated with financial challenges.5,6 XLH does not just affect
individuals with the disease, but also their family and friends,
who are often involved in their support and care.6
Responding to NICE’s final recommendation:
Oliver Gardiner, Trustee and Co-Founder of XLH UK, said: “This
is fantastic news for adults living with XLH in England and Wales,
for whom treatment options are currently severely limited and who,
as a result of this recommendation, will have access to an
effective treatment."
Dr Gavin Clunie, Consultant Rheumatologist and Metabolic Bone
Physician at Addenbrooke’s Hospital, said: “It is welcome that NICE
have decided to recommend burosumab for the treatment of adults
living with XLH, given the clinical benefit to these patients and
significant unmet need. This is a milestone development for those
adults living with XLH who need this treatment.”
Emma Claeys, General Manager UK, Kyowa Kirin, said: “We are
pleased that, following positive engagement with NICE and feedback
from the patient and clinical community, this positive outcome
demonstrates Kyowa Kirin’s commitment to delivering access to
innovative treatments to people living with rare diseases in the
United Kingdom.”
Burosumab is currently the only licensed medicine that addresses
the underlying causes (pathophysiology) of XLH, rather than only
the symptoms. The drug is now reimbursed in several European
countries for both the paediatric and adult populations, including
France, Germany, Italy and Spain.7,8,9
You can learn more about Kyowa Kirin International at:
https://international.kyowa-kirin.com
To view the full press release, click here.
KKI/UK/XLH/0628 Date of preparation: June 2024
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version on businesswire.com: https://www.businesswire.com/news/home/20240621531623/en/
Contacts for Kyowa Kirin Media Stacey Minton:
stacey.minton@kyowakirin.com Victoria Hayes:
victoria.hayes@kyowakirin.com