Three SMA mechanisms are formulary-listed in GCC, and newborn screening is generating 60-80 new gene-therapy candidates a year.

SMA incidence in GCC is estimated at 1 in 6,000 to 8,000 live births, well above the global 1 in 10,000 rate, driven by consanguinity raising the frequency of homozygous SMN1 deletion. Saudi Arabia added SMA to its national newborn-screening programme in 2021, among the first GCC states to do so, with the UAE and Qatar following by 2023; screening coverage reached an estimated 90% of Saudi births, 85% in the UAE, and 75% in Qatar by mid-2024, while Oman, Bahrain, and Kuwait remain below 50%. Newborn-screening-identified pre-symptomatic infants are now being treated at KAMC, KFSH&RC, Sidra Medicine, and SKMC.

All three SMA mechanisms are registered and accessible across GCC. Onasemnogene abeparvovec (Zolgensma), SFDA-registered in 2021, is NPHC-covered for Type 1 and pre-symptomatic newborn-screening-identified infants; NPHC and MOH UAE have negotiated outcomes-based rebate arrangements tied to a 24-month motor-milestone, bringing GCC list price to an estimated $1.5-1.8 million versus $2.125 million in the US. An estimated 60-80 Zolgensma cases occur across GCC annually. Nusinersen (Spinraza), SFDA-registered since 2017, remains standard of care for older, symptomatic Type 2/3 patients, while risdiplam (Evrysdi) is gaining share among families who prefer a daily oral to intrathecal dosing.

Outcomes-based rebates now decide Zolgensma's real GCC price, not the list price alone.

Five questions this report answers:

Q1 - What are the terms of the NPHC outcomes-based rebate arrangement for Zolgensma?

Q2 - How does SMA newborn-screening coverage vary by GCC country, and what does the gap mean for Zolgensma volume?

Q3 - What is driving the shift from nusinersen to risdiplam among older Type 2/3 patients?

Q4 - How does newborn screening shape Zolgensma volume across the six GCC states?

Q5 - How is the 24-month motor-milestone assessed and enforced under the rebate contract?

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Mike || Global Pharma Commercial Marketing Head

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