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NPUK Statement: NICE Olipudase Alfa Decision

Posted: 06.03.24

Yesterday, we were greatly disappointed to learn that the UK’s National Institute for Health and Care Excellence (NICE) has decided 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 means that patients with ASMD type B or type AB in England and Wales will not be able to access this drug via the NHS. This is in stark contrast to Scotland, where the drug is available following preliminary assessment through the Scottish Medicines Consortium (SMC) ultra-orphan framework.

This decision is devastating and will cause significant distress for the ASMD patient and carer community, who have been following and contributing to the development of this treatment for over 20 years.

As a patient community we cannot help but question this decision and the failures of the process that led to it. The clinical evidence speaks for itself, as do decisions made by other regulatory bodies regarding approval and reimbursement of this highly effective technology.

The unmet medical need of ASMD patients in England and Wales is high, with this decision there remains no treatment options except supportive care, which for ASMD, a  multisystemic and progressive disease is both complex and costly.

For our community, this raises many questions, it  highlights the inequality in access to treatments that people affected by ultra-rare conditions experience, an issue at the heart of the Rare Disease Day campaign only last week.

In the coming weeks, NPUK will be working to challenge this decision through the appropriate processes.  To raise your questions and concerns, contact our CEO, Toni Mathieson –

You can read NICE’s final draft guidance, entitled Olipudase alfa for treating Niemann-Pick disease types A and B’, here.