Your Monthly Medicines Roundup for November 2024

In our latest monthly medicines roundup, we do not have any updates on newly approved medicines for inherited metabolic disorders. We do have an update about two newly selected medications for assessment for IMDs, three status updates and one medication that has moved to “not selected”. 

You can find details below: 

Govorestat for treating people aged 2 to 65 years old living with classic galactosemia was selected for assessment by NICE In England. Timelines for the next steps of this assessment are currently unclear. We will provide updates as we receive them.

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Evinacumab for treating children aged 5 to 11 years with homozygous familial hypercholesterolemia was moved to “not selected” by NICE in England. This is on the back of the positive recommendation of evinacumab for people aged 12 years and older living with homozygous familial hypercholesterolemia (see September 2024 roundup). As the assessment for children would be a licence extension, NHS England will be responsible for commissioning this.

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The NICE (England) assessment of triheptanoin for treating people living with a long chain fatty acid disorder (CACT, CPTI, CPTII, LCHAD, TFP, VLCAD) was started with a consultation on the suggested remit, draft scope and provisional stakeholder list. MSUK contributed by submitting a response to the scope consultation. A workshop to discuss responses is expected in December 2024. Further details can be found here:

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The second NICE committee meeting (England) for fosdenopterin, for the treatment of people living with molybdenum cofactor deficiency type A, was held in November. The final outcome of the assessment is currently expected in January 2025, but may be postponed.

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The third NICE committee meeting (England) for olipudase alfa, for the treatment of people living with Niemann-Pick disease types A and B, was held in November. It is currently unclear when the final outcome of the assessment can be expected.

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Thymidine kinase 2 deficiency – Doxecitine–doxribtimine for treating people of any age who had symptoms of thymidine kinase 2 deficiency before 13 years of age was selected for assessment by NICE In England. Timelines for the next steps of this assessment are currently unclear. We will provide updates as we receive them.

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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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