Since 2019, cerliponase alfa (Brineura®) has been provided for eligible NHS patients under a Managed Access Agreement (MAA). With the support of NICE, NHS England and BioMarin have agreed a six-month extension of the current MAA. The extended MAA will now run until 27 May 2025.
All parties will now use the extension period to try to reach an agreement that secures permanent access for current patients and allows new patients to start treatment. During this period, newly diagnosed patients can be assessed for treatment eligibility and start treatment under the terms of the current MAA.
There are currently no arrangements to enable access to cerliponase alfa as part of standard NHS care following the expiry of the MAA. As part of the ongoing NICE evaluation (review of HST12) [ID6145] the next steps will aim to enable commercial discussions between NHS England and BioMarin to progress further and will not affect continuation of treatment under the now extended MAA.
Discussions between NICE, NHS England and BioMarin so far have been constructive, leading to the agreement of the six-month extension, and will now continue. All parties are committed to providing updates to stakeholders, notwithstanding the necessity that discussions are undertaken in a confidential setting.