Pegunigalsidase's TA915 recommendation gives NICE a third Fabry option for the 80-120 UK patients failing standard ERT.

NHS Highly Specialised Services commission Fabry Disease management at lysosomal storage disorder centres (GSTT/King's, Manchester, Addenbrooke's, Birmingham, Edinburgh, Belfast), covering an estimated 800 diagnosed UK patients. Agalsidase beta (Fabrazyme) remains the dominant ERT with roughly 600 patients on therapy; it has never gone through a formal NICE technology appraisal and is instead commissioned directly by NHS England under clinical policy. Migalastat (Galafold, NICE HST4, 2017) offers an oral alternative for the subset with an amenable GLA mutation, confirmed via HEK cell assay at NHS genetics laboratories, with roughly 200 UK patients on oral therapy, an estimated 25% of diagnosed patients.

NICE HST4 (2017) is one of the earliest complete positive HTA decisions for migalastat globally, roughly eight years ahead of most lower-income markets, reflecting a mature and well-structured UK access pathway. The most recent commercial event is pegunigalsidase alfa (Elfabrio, Chiesi/Protalix), a PEGylated ERT positioned for the 80-120 UK patients showing suboptimal response to agalsidase beta: anti-drug antibody positivity, inadequate GL-3 clearance, or continued organ progression. Its NICE appraisal has now concluded as TA915, recommended with an agreed commercial arrangement already in place.

A third Fabry option now exists for patients failing standard enzyme therapy.

Five questions this report answers:

Q1 - What NICE HST4 criteria route UK Fabry patients to migalastat versus enzyme replacement?

Q2 - What cost-effectiveness evidence let pegunigalsidase clear its NICE appraisal (TA915) with a PAS?

Q3 - Which of the 6 NHS lysosomal centres are driving early pegunigalsidase adoption?

Q4 - Why does oral migalastat reach only about 25% of diagnosed UK Fabry patients?

Q5 - Why has agalsidase beta, the dominant ERT, never gone through formal NICE appraisal?

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