Your Monthly Medicines Roundup for August 2024

Metabolic Support UK is delighted to share that during the month of August, one medicine for an inherited metabolic disorder was approved by the National Institute for Health and Care Excellence (NICE) in England.

Additionally, we found out about an update for a medicine of an inherited metabolic disorder by the All Wales Medicines Strategy Group (AWMSG). Finally, we understand that the assessment of one medicine for an inherited metabolic disorder was discontinued by NICE in England.

You can find details below: 

Venglustat for treating gangliosidoses in people 2 years and over. In June we shared that this had been identified by NICE in England in August 2023, with an update from Sanofi in May 2024. A further update was shared end of July which detailed that the clinical trial of venglustat for the treatment of gangliosidoses was discontinued based on the absence of positive trends in outcomes. Based on this, the company will no longer pursue approval in England.

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Alglucosidase alfa in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency). In 2006, this medicine was recommended for restricted use in NHS Wales for infantile and juvenile onset Pompe disease. At the request of several parties a reassessment was conducted by AWMSG in May 2024. Alglucosidase alfa is now recommended as an option for use within NHS Wales for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Pompe disease (acid α-glucosidase deficiency).

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Burosumab for treating X-linked hypophosphataemia in adults. The final recommendation for this medicine has now been published and means it is approved by NICE for the use in England.

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For more updates throughout the medicine approval pathway, check out our Medicines Consultations page by clicking the button below:

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