NICE endorses only approved treatment for ultra-rare Wolman disease for children diagnosed under the age of two: Interview Opportunity
Sebelipase alfa (Kanuma®▼) recommended by NICE as the first treatment for infants with ultra-rare Wolman Disease
Kanuma, an enzyme replacement therapy, is the first medicine to be recommended for this life-threatening condition in infants under two years old
Uxbridge, UK, 27 November 2023: Alexion’s sebelipase alfa (Kanuma®▼), an enzyme replacement therapy, has been recommended by the National Institute for Health and Care Excellence (NICE) for use by the National Health Service (NHS) England for the treatment of Wolman Disease in children who are diagnosed at two years or younger.1
Wolman Disease is a rapidly progressing and life-threatening disease that causes multi-organ damage. Symptoms in newborns or very young infants include enlarged liver and spleen, poor weight gain, low muscle tone, jaundice, vomiting, diarrhoea, developmental delay, anaemia, and poor absorption of nutrients from food. Infants with Wolman Disease normally do not live to see their first birthday without treatment.2
In the final draft guidance published today, NICE recognised the importance of sebelipase alfa as a life-saving treatment option for people with Wolman disease. The guidance also specified that the recommendation is not intended to affect treatment with sebelipase alfa that was started in the NHS before this guidance was published.[i] People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop. This decision should be made jointly by the clinician, the child, and their parents or carers.1
Professor Simon Jones, Consultant in Paediatric Inherited Metabolic Disease, Manchester University NHS Foundation Trust (MFT) said: "Having shown in clinical trials at the NIHR Manchester Clinical Research Facility at Royal Manchester Children’s Hospital (part of MFT) that we could treat a fatal disease of infancy with this lifesaving drug is extremely gratifying. Moving forward to treat children on the NHS is however what we all really want and signifies a substantial step forward in our dedication to practical advancements in rare disease medicine and improved patient outcomes.”
Sean Richardson, Vice President and General Manager, Alexion, UK: “Today is a milestone moment for infants born with Wolman Disease. Ensuring access to new treatments for rare diseases is essential – it is not just about medicine; it is about giving everyone, no matter how rare their condition, the possibility of improved health.
The recommendation is the result of continued constructive collaboration with NICE, NHS England, and other important stakeholders to establish a sustainable access solution for this ultra-rare disease, but we must find ways to ensure all parties can more swiftly deliver lifesaving medicines to this underserved patient group.”
Bob Stevens, Group Chief Executive of the MPS Society, said: “Today’s announcement is positive news, giving babies born with Wolman Disease and their parents access to a new treatment option. It remains challenging to get medicines for rare diseases reimbursed but our commitment to our community of MPS and rare disease families is unwavering. Moments like this are special and are only possible through a collaborative process where the patient voice is heard, valued, and acted upon.”
ENDS
About Wolman Disease
Wolman Disease is a progressive, ultra-rare metabolic disease which occurs when deficient LAL enzyme activity leads to the continuous, uncontrolled accumulation of lipids in vital organs such as the liver, blood vessels and other tissues. In infants, Wolman Disease is a rapidly progressive disease that often results in liver failure and premature mortality within 12 months of age.[ii]
About KANUMA® (sebelipase alfa)
KANUMA® (sebelipase alfa) is an innovative enzyme replacement therapy that treats the underlying cause of lysosomal acid lipase deficiency (LAL-D). The medication works by replacing the deficient lysosomal acid lipase (LAL) enzyme and reducing the accumulation of fatty materials in the lysosomes of cells throughout the body. KANUMA is administered intravenously. KANUMA is approved for the treatment of LAL-D in the United States (U.S.), European Union (EU), Japan, Canada and other countries around the world.[iii]-[iv]
About Alexion
Alexion, AstraZeneca Rare Disease, is the group within AstraZeneca focused on rare diseases, created following the 2021 acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare diseases for nearly 30 years, Alexion is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialisation of life-changing medicines. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on haematology, nephrology, neurology, metabolic disorders, cardiology and ophthalmology. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. Please visit alexion.com/worldwide/UK.
About AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on social media @AstraZeneca.
About Manchester University NHS Foundation Trust
Manchester University NHS Foundation Trust is the largest NHS Trust in the country and a leading provider of specialist healthcare services. Its ten hospitals are home to 28,000 staff including world class clinicians and academic staff committed to finding patients the best care and treatments. Its 10 hospitals are Manchester Royal Infirmary, Saint Mary's Managed Clinical Service, Royal Manchester Children's Hospital, Manchester Royal Eye Hospital, University Dental Hospital of Manchester, Trafford General Hospital, Altrincham Hospital, Wythenshawe Hospital, Withington Hospital and North Manchester General Hospital. More information is available at www.mft.nhs.uk.
About the NIHR
The mission of the National Institute for Health and Care Research (NIHR) is to improve the health and wealth of the nation through research. We do this by:
- Funding high quality, timely research that benefits the NHS, public health and social care;
- Investing in world-class expertise, facilities and a skilled delivery workforce to translate discoveries into improved treatments and services;
- Partnering with patients, service users, carers and communities, improving the relevance, quality and impact of our research;
- Attracting, training and supporting the best researchers to tackle complex health and social care challenges;
- Collaborating with other public funders, charities and industry to help shape a cohesive and globally competitive research system;
- Funding applied global health research and training to meet the needs of the poorest people in low and middle income countries.
NIHR is funded by the Department of Health and Social Care. Its work in low and middle income countries is principally funded through UK Aid from the UK government.
[i] National Institute for Health and Care Excellence. (2023). Sebelipase alfa for treating Wolman disease [Final draft guidance]. https://www.nice.org.uk/guidance/gid-hst10047/documents/674 Last Accessed Nov 2023.
[ii] Cossette, A et al. (2022). Early diagnosis and successful long-term management of a rare, severe lysosomal acid lipase deficiency/Wolman disease patient: Infancy to age five. Can Liver Journal; 5(3): 428-434. Early diagnosis and successful long-term management of a rare, severe lysosomal acid lipase deficiency | Canadian Liver Journal (nih.gov). Last Accessed Nov 2023.
[iii] European Medicines Agency. (2023) Kanuma Summary of Product Characteristics. https://www.ema.europa.eu/en/medicines/human/EPAR/kanuma Last Accessed Nov 2023.
[iv] FDA. Kanuma Prescribing information. https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/125561s013lbl.pdf Last accessed Nov 2023.
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