Your Monthly Medicines Roundup for February 2024

Your Monthly Medicines Roundup for February 2024

Well, February’s done, it’s nearly spring and hopefully some warmer weather!

It has been a quiet month in terms of public announcements for newly approved metabolic medicines. That does not mean that we have been waiting quietly for March to roll in. For many newly approved metabolic medicines, we work with AWSMG, Health NI, NICE and SMC for several months, sometimes years, in the lead up to the approval. 

We are currently working on, or providing support on, the approval pathways for the following medicines, alphabetised by disorder name: 

Arginase 1 deficiency

Pegzilarginase for treating arginase-1 deficiency is currently being accessed by NICE in England under their highly specialised technology pathway. 

For further details, click the button below:

Molybdenum Cofactor Deficiency Type A

Fosdenopterin for treating molybdenum cofactor deficiency type A is currently being accessed by NICE in England under their highly specialised technology pathway.

For further details, click the button below:

X-linked hypophosphatemia

Burosumab for treating X-linked hypophosphataemia in adults is currently being accessed by NICE in England under their standard technology pathway.

For further details, click the button below:

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