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NPUK Frequently Asked Questions (FAQs) | NICE Xenpozyme (Olipudase Alfa) Decision

Posted: 09.04.24

What is the NICE Xenpozyme (Olipudase Alfa) Decision?

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This decision outlined by NICE in their Final Draft Guidance means that ASMD type AB or type B patients in England and Wales will not be able to access olipudase alfa through the NHS.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

Acid sphingomyelinase deficiency (ASMD) (historically known as Niemann-Pick disease types A, A/B, and B,) is an ultra-rare, inherited disease.  ASMD is progressive and life-limiting and significantly reduces life expectancy. The severity of symptoms causes multiple long-term health issues and prevent patients from fully participating in daily activities.

What is Xenpozyme (Olipudase Alfa)?

Olipudase Alfa has been designed to replace the enzyme that is lacking in people with ASMD. Olipudase Alfa acts in the same way as the natural ASM enzyme would, and therefore acts as a replacement, reducing the build-up of sphingomyelin in the organs and treating the signs and symptoms of the disease. It is used to treat the signs and symptoms of ASMD that are not related to the brain, and it is suitable for children and adults diagnosed with ASMD type A/B or B.

Why is Xenpozyme (Olipudase Alfa)  available in Scotland and not in England or Wales?

In England and Wales, the NICE highly specialised technology (HST) programme, evaluates medicines for very rare conditions. In Scotland, the Scottish Medicines Consortium (SMC) uses different criteria, methodologies and decision-making processes for appraising ultra-orphan medicines under its ultra-orphan assessment framework, leading to different outcomes and variations in access to ultra-orphan medicines across the UK.

I currently have access to Xenpozyme (Olipudase Alfa), will this continue?

NICE’s Final Draft Guidance document recommends that patients are not taken off the drug if they are benefitting clinically. The company behind olipudase alfa, Sanofi, has agreed to continue supply of the drug for patients in England and Wales who are already enrolled in their compassionate use programme or for those who were part of the clinical trial. However, as this does not include funding for clinical monitoring or homecare, it is down to the patient’s individual hospital trust as to whether access will continue. Please contact us if you experience any issues, we are here to support you and to advocate on your behalf.

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 template letters below and our top tips for how to construct an effective letter. You can find your local MP using www.theyworkforyou.com Please don’t forget to copy us into any correspondence – just add our email info@npuk.org to the ‘cc’ line.  This is 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