Question to the Department of Health and Social Care:

To ask the Secretary of State for Health and Social Care, what steps his Department is taking to make cerliponase alfa available to eligible children in advance of an agreement being signed between NHS and the manufacturer of the drug.

The National Institute for Health and Care Excellence (NICE) is currently evaluating cerliponase alfa for use in the treatment of neuronal ceroid lipofuscinosis type 2, through its highly specialised technologies programme. NICE published draft guidance on cerliponase alfa in February 2018 that does not recommend use of the drug as there is considerable uncertainty about the long-term effects of the drug.

NHS England is in discussions with Biomarin, the manufacturer of cerliponase alfa, to seek to agree a managed access agreement (MAA) that may address the uncertainty and enable NICE to recommend the drug as value for money. Cerliponase alfa is not therefore currently routinely funded on the National Health Service, but NHS England and the company are working jointly to agree a MAA.

In the absence of final guidance from NICE, clinicians can apply to NHS England for funding in exceptional cases through the individual funding request process.