Your Monthly Medicines Roundup for April 2024

Your Monthly Medicines Roundup for April 2024

It has been another quiet month in terms of public announcements for newly assessed metabolic medicines. We know that a decision on at least one metabolic medicine will be announced next month, and we hope there will be more.

In the meantime, there are several metabolic medicines currently going through the approval processes of AWSMG, Health NI, NICE and SMC, which are being supported by our patient organisation partners, including, in alphabetical order: 

Adrenoleukodystrophy

Adrenoleukodystrophy (ADL) Leriglitazone for treating andrenoleukodystrophy is currently being assessed by NICE in England under their standard technology pathway. Alex – The Leukodystrophy Charity are providing the lived experience to this submission. 

For further details, click the button below:

Neuronal Ceroid Lipofuscinosis-Type 2

Neuronal Ceroid Lipofuscinosis-Type 2Cerliponase alfa for treating CLN2, a form of Batten disease, is currently being assessed by NICE in England under their standard technology pathway. Batten Disease Family Association are providing the lived experience to this submission.

For further details, click the button below:

Fabry

FabryPegunigalsidase alfa for treating Fabry disease is currently being assessed by SMC in Scotland as a resubmission. MPS Society are providing the lived experience to this submission.

For further details, click the button below:

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