All three UK SMA therapies cleared NICE with confidential pricing, so the 2021 newborn screening programme now decides who gets treated first.

An estimated 1,000 UK patients live with spinal muscular atrophy across all types. The UK added SMA to its national newborn screening programme in 2021, the first such programme in Europe, identifying roughly 25 pre-symptomatic patients per year and routing them to one of 6 specialist centres. NICE has recommended all three approved SMA therapies through confidential Patient Access Scheme agreements: Zolgensma, via the Highly Specialised Technology route (HST15, 2021, expanded to pre-symptomatic patients under HST24); Spinraza (TA588, 2019), an intrathecal option for Types 1-3; and Evrysdi (TA755, 2022), an oral SMN2 modifier families increasingly prefer.

The NHS treatment algorithm is now type- and age-based rather than access-based: Type 1 and NBS-identified pre-symptomatic patients under 2 years route to Zolgensma; Type 2/3 patients choose between nusinersen and risdiplam. All three agents carry confidential PAS pricing, so relative NHS net cost is commercially sensitive, but Zolgensma's cost-effectiveness case for its $2.125 million list price was only accepted under a confidential commercial arrangement negotiated through the HST15 appraisal, later expanded to presymptomatic infants under HST24. Nusinersen remains the default for roughly 300 UK patients, largely adults outside Zolgensma's age/weight eligibility, while risdiplam's oral dosing is winning share by reducing intrathecal procedure burden.

Access is resolved for all three; family and physician preference now decides share.

Five questions this report answers:

Q1 - How is the 2021 newborn screening programme changing first-prescribing decisions for Zolgensma?

Q2 - What confidential PAS pricing structure let Zolgensma's $2.125 million list price clear NICE?

Q3 - Why are UK families increasingly choosing oral risdiplam over intrathecal nusinersen for Type 2/3?

Q4 - How did HST24 expand Zolgensma's NICE recommendation to pre-symptomatic infants in 2023?

Q5 - Why does nusinersen remain the default for roughly 300 UK adult patients?

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