Migalastat is covered for 35-50% of GCC Fabry patients with an amenable mutation, but fewer than 15% have been tested.

Classic Fabry disease prevalence in GCC is estimated at 1 in 20,000 to 30,000 males, elevated above the global 1 in 40,000 rate by founder mutations concentrated in consanguineous families. The KFSH&RC Fabry registry has documented more than 15 unique GCC family clusters sharing GLA mutations, and family-based cascade screening at index-case diagnosis yields an estimated 3 to 5 additional affected members per family, nearly triple the 1.8 seen in global series. Agalsidase beta (Fabrazyme), SFDA-registered since 2003 and covered by NPHC, is the dominant enzyme replacement therapy, administered at KAMC, KFSH&RC, AUH, and Hamad Medical Corporation.

GCC is structurally a two-ERT market in a way the US is not: agalsidase alfa (Replagal), EMA-approved but never FDA-approved, reaches GCC patients via SFDA's EMA-pathway registration and Takeda's regional distribution, with some NPHC-approved patients maintained on it during Fabrazyme global supply constraints, as occurred in 2010 and 2022. Migalastat (Galafold), the oral chaperone SFDA-registered in 2020, is covered for the estimated 35-50% of patients carrying an amenable GLA mutation, but eligibility requires a HEK293 cell-based assay available at only one laboratory in the region, KFSH&RC, with a 6-8 week turnaround from elsewhere in the Gulf.

Mutation-testing access, not clinical evidence, is the binding constraint on oral-therapy uptake.

Five questions this report answers:

Q1 - How does the two-ERT structure affect switching decisions for GCC Fabry patients?

Q2 - What would it take to expand HEK293 testing beyond KFSH&RC's single lab?

Q3 - How is family-based cascade screening identifying new Fabry patients across GCC genetics centres?

Q4 - Why hasn't migalastat captured its full amenable-mutation population in the GCC?

Q5 - What GLA mutation testing turnaround time do Gulf patients face outside KFSH&RC?

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Mike || Global Pharma Commercial Marketing Head

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