Since 2019 cerliponase alfa (Brineura®) has been provided for eligible NHS patients under a Managed Access Agreement (MAA). On 9 October 2024, with the support of NICE, NHS England and BioMarin agreed a six-month extension of the current MAA to run until 27 May 2025. On 2 April 2025, a further update was provided on an agreement in principle for an additional three-month extension to the current MAA, to run until 27 August 2025.
Following the third NICE Committee on 3 April 2025, an extension to the current MAA under existing terms will be put in place until the end of December 2025 or final NICE guidance publication if earlier, providing the opportunity to continue work towards a long-term agreement with the aim of enabling comprehensive access for all eligible patients.
In advance of that, NHS England can confirm that it has reached an agreement with BioMarin that secures permanent access to this treatment for all patients initiated on treatment at the point the MAA ends.
In addition, during this further extension period of the MAA, newly diagnosed patients can still be assessed for eligibility and start treatment.
Discussions between NICE, NHS England and BioMarin have remained constructive, already enabling permanent access to this treatment for current patients to be secured and leading to a further extension of current MAA arrangements that provides additional time for commercial negotiations relating to newly diagnosed patients to continue.
All parties remain committed to providing updates to stakeholders, notwithstanding the necessity that discussions are undertaken in a confidential setting.