Inequality in UK access to Xenpozyme infuriates Sanofi

The UK's National Institute for Health and Care Excellence (NICE) has decided not to recommend Sanofi's (Euronext: SAN) 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 type AB or type B ASMD in England and Wales cannot access via the National Health Service (NHS) what is the only licenced treatment for this progressive, life-limiting, ultra-rare disease, which affects a very small number of children and predominantly young adults.

"Patients with ultra-rare diseases in England and Wales are being let down"In contrast, the Scottish Medicines Consortium has made Xenpozyme available via its ultra-orphan pathway, meaning that eligible patients can access the drug while additional data is collected for three years.