Since 2019 cerliponase alfa (Brineura®) has been provided for eligible NHS patients under a Managed Access Agreement (MAA).
Following a series of extensions to the MAA for cerliponase alfa through 2024 and 2025, NHS England and BioMarin, supported by NICE, have agreed an additional extension to the current MAA.
The extension will be under the same terms as the existing MAA and will run until the end of June 2026, or the publication of final NICE guidance if earlier.
NHS England has already confirmed 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 or at publication of final NICE guidance. In addition, during this further extension period of the MAA, newly diagnosed patients can still be assessed for eligibility and start treatment.
Next steps will be subject to the outcome of the appeals being heard on 25 November and we will update stakeholders in due course.