Sanofi’s Xenpozyme (olipudase alfa) enzyme replacement therapy has not been recommended by the National Institute for Health and Care Excellence (NICE) to treat acid sphingomyelinase deficiency (ASMD) types AB or B.
Also known as Niemann-Pick disease, ASMD is an ultra-rare lysosomal storage disorder that results from a deficiency of the enzyme acid sphingomyelinase.
An estimated 40 to 50 people in England are living with the disease, which predominantly affects young adults and causes symptoms including fatigue, organ enlargement, poor growth, susceptibility to respiratory infections and bone pain.
There are no licensed treatments for the underlying causes of ASMD, NICE outlined in its final draft guidance for Xenpozyme, adding that best supportive care is currently used to manage the symptoms of the disease.
The agency noted that clinical evidence showed Xenpozyme was associated with improvements in lung function and reductions in spleen size in adult and paediatric ASMD patients after one year of treatment, with benefits potentially continuing in the longer term and becoming more gradual as the condition stabilises.
It decided, however, that there were “uncertainties in the economic model” for the therapy and that available cost-effectiveness estimates were higher than what it usually considers “an acceptable use of NHS resources for highly specialised technologies”.
The rejection contrasts with the Scottish Medicines Consortium’s decision to make Xenpozyme available via its ultra-orphan pathway, allowing eligible ASMD patients to access the therapy while additional data is collected.
Rippon Ubhi, country lead for Sanofi UK and Ireland, described NICE’s decision as “devastating news for the ASMD community”, adding that it created “inequity in access within the UK”.
Ubhi said in a statement that the company had proposed “a number of commercial arrangements” to support access to Xenpozyme but that “no solution could be found within the constraints of the existing framework”.
She added: “We are keen to have the opportunity to engage with [the] government, NICE and NHS England to urgently discuss and adapt the way that innovative treatments for ultra-rare conditions are assessed, to ensure that patients in England and Wales can access the same potentially life-changing treatments as those living elsewhere in the UK, and in other countries.”