Managed Access Agreement resources

For patients and clinicians

Vimizim Treatment for Morquio A – The Managed Access Agreement

Treatment for Morquio A in England with enzyme replacement therapy Vimizim® (elosulfase alfa) is made available through a Managed Access Agreement.

We have produced this guide, which explains how this process of obtaining and staying on treatment works.

This patient guide is available for download in English, Urdu, Pashto and Turkish.

Brineura Treatment for CLN2 Disease – The Managed Access Agreement

Brineura® (cerliponase alfa) is an enzyme replacement therapy for the treatment of CLN2 disease (Batten disease). It is the first disease-modifying treatment available for CLN2 disease, which was authorised for use in 2017. It is currently available in England through a Managed Access Agreement (MAA).

This guide helps explain the MAA, the criteria set out in it to allow a child to start treatment with Brineura and how treatment is monitored during the agreement.

This parent/carer guide is available for download in English and Polish.

Further Advice

You can find further details for both Vimizim and Brineura treatments on the NICE website.

If you are a patient or parent/carer for someone on one of the above Managed Access Agreements and need more guidance please contact your consultant.

Further support can be offered by your patient organisation, please refer to the final page on the Managed Access Agreement guide for contact details.