Your Monthly Medicines Roundup for June 2024

Your Monthly Medicines Roundup for June 2024

It has been another quiet month in terms of public announcements for newly assessed metabolic medicines.

Draft decisions were published by NICE for Olipudase alfa for treating Niemann-Pick type AB or type B and Burosumab for treating X-linked hypophosphataemia in adults. While we wait for the final NICE guidance to be published, we would like to give you an update about medicine which have been identified by NICE for review. Review by NICE often means review by AWSMG, Health NI and SMC will soon follow, or they will adopt NICE’s recommendation: 

Gaucher Disease Type 3

Venglustat for treating type 3 Gaucher disease in people aged 12 and over after at least 3 years of enzyme replacement therapy. This was identified by NICE in April 2023, with an update from Sanofi in May 2024. No final timelines have been set.

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Gangliosidoses

Venglustat for treating gangliosidoses in people 2 years and over. This was identified by NICE in August 2023, with an update from Sanofi in May 2024. No final timelines have been set.

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Glycogen storage disease type 1a

Pariglasgene brecaparvovec for treating glycogen storage disease type 1a in people 8 years and over. This was identified by NICE in June 2023. No update has been provided since.

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Hyperoxaluria type 1

Nedosiran treating primary hyperoxaluria type 1 in people 6 years and over. This was identified by NICE in March 2023. No update has been provided since.

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LC-FAOD

Triheptanoin for treating long-chain fatty acid oxidation disorders. This was identified by NICE in November 2023, with an update from Ultragenyx in April 2024. Based on information provided we estimate that the appraisal will start in February 2025.

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X-linked andrenoleukodystrophy

Leriglitazone for treating X-linked andrenoleukodystrophy. This was identified by NICE in August 2023. No update has been provided since.

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X-linked protoporphyria

Dersimelagon for treating erythropoietic protoporphyria and X-linked protoporphyria in people 12 years and over. This was identified by NICE in July 2022. No update has been provided since.

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