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NPUK launches campaign challenging decision to not recommend the use of Xenpozyme (Olipudase Alfa) for ASMD Niemann-Pick disease type AB or type B

Posted: 05.04.24

In March 2024, the National Institute for Health and Care Excellence (NICE) announced their decision not to recommend Sanofi’s Xenpozyme (olipudase alfa) as a treatment for acid sphingomyelinase deficiency (ASMD) Niemann-Pick disease in people with type AB or type B.

This decision outlined by NICE in their Final Draft Guidance means that ASMD type AB or type B patients in England and Wales will not be able to access olipudase alfa through the NHS.

As a result, there will be no treatment options for patients except best supportive care.  Best supportive care is focused on managing symptoms and helping to keep patients as well as possible when there are no specific or disease modifying treatment options available. For ASMD, best supportive care is both complex and costly and does not prevent disease progression.

What action is NPUK taking in response to this decision?

NPUK want to see effective treatments approved for use in all types of Niemann-Pick diseases, at prices that are affordable for our health system, but with the appropriate flexibility within that system to  take into account the highly complex nature of developing medicines for our ultra-rare diseases.

As formal consultees, we have been involved in the NICE appraisal process from the outset, and in the weeks since this decision was announced, we have taken steps to appeal.  Through our advocacy efforts, we have secured an appeal hearing, which is scheduled to take place on Friday 24 May.

In addition, we will be writing to a number of key stakeholders to express our concern on behalf of ASMD patients in England and Wales, and to highlight the severe unmet medical needs of this community.

We will continue to do all we can, through the appropriate channels, to influence change and we will keep the community informed of our progress.

What can I do to help?

With your help, our voice can be louder and stronger. Together we can ensure that news of this decision, and the impact for patients and families, does not go unrecognised.

Sign up to our campaign by email.  John Lee Taggart, NPUK Head of Communications, will keep you up to date with our campaign progress and relevant news.

Write to your MP. it’s easier than ordering a takeaway and it’s also the most effective way to make your voice heard. See our dedicated page here which includes our top tips on how to construct an effective letter.

You can find your local MP using www.theyworkforyou.com

Please use the following template letter(s) as a guide for your own – remember that your letter will have more of an impression if you add your own personal experiences/feelings on the subject, so please feel free to edit the wording, or simply write your own following our format.

Write to your MP, Template (Xenpozyme Olipudase Alfa)

To support your MP in raising your concerns as quickly and efficiently as possible, we have developed template letters to support them in communicating with colleagues and other key stakeholders in relation to the Olipudase Alfa decision. Once you have established contact, simply complete the appropriate template and send it to your MP.  You may wish to ask them to write to someone specifically, who you feel could help to address your concerns.

Template for MPs – Children (Xenpozyme Olipudase Alfa)
Template for MPs (Adults, Xenpozyme Olipudase Alfa)

Please don’t forget to copy us into any correspondence – just add our email info@npuk.org to the ‘cc’ line.  This is to help us to understand the support we have for the campaign and follow up as needed.

Get Social. Engage with our social media activity on this subject and help to ensure that our message reaches a wider audience. Please like, comment and share items from our official NPUK pages.

If you or someone you know is directly affected by this decision and you would like to help us highlight the true impact for patients and their families, please get in touch with John – we would love to hear from you and to help you to share your quotes and personal stories in line with this campaign.

If you have any questions, please don’t hesitate to reach out to us by email: info@npuk.org or by phone: 0191 415 06 93