LONDON--(BUSINESS WIRE)--Adults living with the rare disease X-linked hypophosphataemia (XLH) in England and Wales risk missing out on an innovative treatment following an initial decision by NICE not to recommend burosumab (marketed as Crysvita) to treat adults who have a confirmed diagnosis of XLH. However, whilst Kyowa Kirin is disappointed with NICE’s decision, outlined in its initial Appraisal Consultation Document (ACD) published today2, it remains committed to finding a solution for adults living with XLH to have access to the medicine and will continue to work with NICE, NHS England and all other relevant stakeholders to find a resolution.
The decision would create a disparity in access between England and Wales on the one hand, and Scotland on the other. Adults living with XLH in Scotland who need it have been able to be treated with Crysvita on the NHS since February 2023, following approval by the Scottish Medicines Consortium.3 This demonstrates that Crysvita can be clinically beneficial and cost effective for this group of patients. To date, ten European countries, including France, Germany and Italy, also reimburse the medicine for the treatment of adults with XLH.
XLH is a very rare genetic condition, affecting roughly 1.7 – 4.8 per 100,000 people in the UK.4 It is a life-long and progressive disease that typically presents in early childhood, causing bowed legs, stunted growth, and bone and joint pain.5 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.6 XLH also has an adverse impact on emotional wellbeing, primarily due to pain, uncertainty about the future, and financial challenges that may also be associated with XLH.7,8 XLH does not just affect individuals with the disease, but also their family and friends, who are often involved in their support and care.7
Crysvita was routed by NICE to its Standard Technology Assessment pathway rather than the Highly Specialised Technology evaluation (HST) pathway, which offers additional flexibilities to support patient access to treatments for very rare diseases. Kyowa Kirin and clinical experts opposed the routing to STA, due to the inevitable challenges that this would create as a result of the different methods of evaluation used.9,10 The appraisal process, which started more than 18 months ago, has been fraught with challenges. However, Kyowa Kirin remains committed to continued engagement with NICE to ensure patients living with XLH are able to access the treatment.
Responding to NICE’s initial decision:
Oliver Gardiner, Trustee and Co-Founder of XLH UK, said: “This decision is a huge blow for adults living with XLH in England and Wales, for whom current treatment options are severely limited and who as a result of this recommendation will miss out on a potentially life-changing treatment.”
Dr Gavin Clunie, consultant rheumatologist and metabolic bone physician at Addenbrookes Hospital, said: “It is disappointing that NICE have decided to not recommend Crysvita for the treatment of symptomatic adults living with XLH in this interim decision, given the clear clinical benefit to these patients and significant unmet need.”
Emma Claeys, General Manager, Northern Cluster, Kyowa Kirin, said: “We are disappointed that, despite feedback from the patient and clinical community, together with extensive evidence provided, the appraisal committee’s interim decision is negative. We fully support the UK Rare Disease Framework and specifically priority 4 - improving access to specialist care, treatments and drugs. This is critically important given that many people with rare diseases, including those with XLH, face challenges to access safe, high-quality care and treatments.
It is vital that NICE reconsider this initial decision in advance of the publication of final guidance to ensure adults who are severely impacted with XLH and their families have the opportunity to live their lives to the full. Kyowa Kirin remains committed to working constructively with NICE to deliver access to patients.”
Crysvita is the only medicine addressing the root causes (pathophysiology) of XLH. As such, there is a high unmet need for adults with XLH whose symptoms currently affect their daily, family, social, and work lives. The treatment is currently available for children and young people living with XLH in all four devolved nations within the United Kingdom. However, following transition from paediatric to adult care, XLH symptoms may persist or reemerge following cessation of treatment.11 Without the option for treatment with Crysvita in adulthood, XLH patients may face further complications from the condition and a poorer quality of life.11
Notes to editor:
About X-linked hypophosphataemia
XLH is caused by a genetic mutation which leads to overexpression of the protein FGF23, a protein involved in the regulation of phosphate concentration in the blood. In XLH, FGF23 is produced in excess leading to depletion of phosphate in the blood, known as hypophosphataemia.12
Individuals living with the disease may display a multitude of symptoms including short stature, limb deformities, bone and joint pain, oral abscesses, and hearing loss.13 To manage this wide variety of symptoms, the disease is managed through multi-disciplinary teams.13
About CRYSVITA® (burosumab) in XLH
Crysvita (burosumab) is a recombinant human monoclonal antibody that binds to the protein FGF23. This has the impact of inhibiting the action of FGF23, allowing phosphate regulation in the body to be restored.14
Phase 3 clinical trial data has demonstrated the safety and efficacy of Crysvita in the treatment of XLH in adult populations.15,16 Considering this, the European Commission granted Crysvita a conditional marketing authorisation in 2020, for the treatment of adolescents regardless of growth status and adults with XLH, which was then upgraded to a full marketing authorisation in 2022.17,18 The drug is now reimbursed in several EU countries for both the paediatric and adult population, including Scotland, France and Germany.3,19,20
Kyowa Kirin and Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE: Ultragenyx) have been collaborating in the development and commercialisation of Crysvita globally, based on the collaboration and licence agreement between Kyowa Kirin and Ultragenyx.
About Kyowa Kirin
Kyowa Kirin strives to create and deliver novel medicines with life-changing value. As a Japan based global specialty pharmaceutical company with a heritage of more than 70 years, the company applies cutting-edge science, including expertise in antibody research and engineering, to address the needs of patients across multiple therapeutic areas such as nephrology, oncology, immunology/allergy and neurology. Across its four regions – Japan, Asia Pacific, North America and EMEA/International – Kyowa Kirin focuses on its purpose, to make people smile, and is united by its shared values of commitment to life, teamwork, innovation and integrity.
You can learn more about Kyowa Kirin International at: https://international.kyowa-kirin.com/uk/
References
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KKI/UK/BUR/0413
Date of preparation: November 2023
