Advances in Therapy· 2026Q1
Real-World Treatment Patterns in Patients with Spinal Muscular Atrophy Receiving Multiple Disease-Modifying Therapies
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- Q1SCImago
- 2026year
Short summary
Following widespread newborn screening, onasemnogene abeparvovec (OAV) is the most common first treatment for spinal muscular atrophy (SMA) in infants (age ≤ 2 years), used by 64.6% of patients initiating therapy in 2022-2024. However, patients initiating OAV as their first therapy were least likely to receive a second disease-modifying therapy (DMT).
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Key points
- In 2022-2024, 64.6% of infants (age ≤ 2 years) with SMA initiated treatment with onasemnogene abeparvovec (OAV).
- Among patients initiating therapy at age ≤ 2 years, 55.6% of those starting with nusinersen received a second DMT, compared to 12.0% of those starting with OAV.
- For patients receiving multiple DMTs, nusinersen was the most common first DMT (75.4%) between 2019-2024, with a median time of 18.9 months to a second DMT.
- In a subgroup of infants (age ≤ 2 years) receiving multiple DMTs, OAV was the most common first DMT, and all patients treated with risdiplam received OAV as their second DMT.
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Abstract
INTRODUCTION: Nusinersen (approved in 2016), onasemnogene abeparvovec (intravenous infusion [OAV], 2019; intrathecal injection, 2025), and risdiplam (2020) are disease-modifying therapies (DMTs) available in the US for spinal muscular atrophy (SMA). Real-world evidence on treatment patterns among patients receiving multiple DMT types remains limited. METHODS: This retrospective study used the Komodo Health Research Database (2016-2024) to identify patients with SMA treated with DMTs. First DMT distribution and the proportion of patients receiving a second DMT were described for patients who initiated any DMT at age ≤ 2 years in 2022-2024 (following widespread newborn screening [NBS]). Patient characteristics, treatment sequences, and time between DMT initiations were described for patients with multiple DMTs whose first was initiated on/after May 2019, including subgroups receiving first DMT at age ≤ 2 years in 2022-2024. RESULTS: Among patients who initiated any DMT at age ≤ 2 years in 2022-2024 (N = 257), 64.6% started with OAV, 24.9% with risdiplam, and 10.5% with nusinersen. Of these, 12.0%, 20.3%, 55.6% of patients treated with OAV, risdiplam, and nusinersen, respectively, received a second DMT. Among patients receiving multiple DMTs during 2019-2024 (N = 341), 75.4% first initiated nusinersen, 15.2% risdiplam, and 9.4% OAV; mean age at first DMT was 15.4 years overall (17.3, 15.2, and 0.8 years, respectively). Median time from first to second DMT was 18.9 months overall. Within this cohort, 44 patients initiated their first DMT at age ≤ 2 years in 2022-2024; OAV was the most common first DMT. All patients treated with risdiplam and most patients treated with nusinersen received OAV as their second DMT; median time from first to second DMT was 13.4 months. CONCLUSIONS: Following widespread NBS, OAV had the highest utilization among patients age ≤ 2 years as first or second DMT after nusinersen or risdiplam. Use of a second DMT type was lowest among patients who initiated OAV.
The authors' abstract, as published at the source. Advances in Therapy, 2026 · DOI ↗
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Field: Genetics (Medicine)
GeneticsMedicine