Press release – NICE condemns to death up to three babies each year in England

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Press release – NICE condemns to death up to three babies each year in England

NICE the drug decision making body have announced their final decision  today, Wednesday 15 February 2017, not to recommend Kanuma (Sebelipase alfa), the treatment for  individuals suffering from Lysosomal Acid Lipase Deficiency (LAL D).

Kanuma was given fast track approval by the EMA in June 2015, following an accelerated assessment which recognised the lack of any other treatment and very high death rate in the infant population. This process was designed to speed up patient access to new medicines where there was an unmet need.

Despite this approval, compelling clinical evidence and clinical practice in the UK, to treat, protect and where possible prevent death, especially in children, NICE have decided not to recommend treatment for both the infant and late onset form of LAL D. This will result in the unnecessary death of up to three children a year under the age of 6 months in England.

The infant form of LAL D (previously known as Wolman’s disease), is a rapidly progressive and aggressive disease that causes severe malabsorption, weight loss and growth failure. Early diagnosis and access to treatment is vital for these children to survive. Without treatment children will die within the first 6 months of life.

Although the disease can be variable for children and adults with the late onset form of the disease, its effects are multi systemic, with some patients developing chronic liver disease requiring a liver transplant.

Christine Lavery, Group Chief Executive of the Society for Mucopolysaccharide Diseases said:

We are particularly devastated by the announcement from NICE to deny babies with LAL D access to life saving therapy. These babies need immediate access to treatment following diagnosis. Without treatment children will unquestionably die a cruel and inhumane death within days or weeks.

It is inconceivable that babies in England can be sacrificed in this way, when the UK Government is so committed to channelling billions of pounds through the International Development Fund to help the world’s most vulnerable.

In the clinical trial Kanuma has given children and adults their life back and hope for a future without long-term disease burden and early mortality and morbidity.

Should a right to life be denied when there is a treatment that not only demonstrates clinical benefit but allows children to thrive, grow, meet their developmental milestones and have a normal childhood.

The MPS Society will continue to work with and on behalf of patients, to ensure access to treatment is available to all those who will benefit.


The NICE decision can be downloaded here.

About the MPS Society
The Society for Mucopolysaccharide Diseases (The MPS Society) is the only registered UK charity providing professional support to over 1200 families in the UK affected by MPS and related disease. The MPS Society provides a pioneering advocacy service to individual families as well as fund innovative and life changing clinical and academic research.

As a registered charity the Society is entirely supported by voluntary donations and fundraising.

Sophie Thomas
Advocacy Support Team Manager
MPS Society

0345 389 9901

Society for Mucopolysaccharide Diseases (MPS Society), MPS House, Repton Place, White Lion Road, Amersham, Buckinghamshire, HP7 9LP

15 February 2017

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