Long-term follow-up of patients with type 2 and non-ambulant type 3 spinal muscular atrophy (SMA) treated with olesoxime in the OLEOS trial

OLEOS Study Group, Francesco Muntoni, Enrico Bertini, Giacomo Comi, Janbernd Kirschner, Anna Lusakowska, Eugenio Mercuri, Mariacristina Scoto, W. Ludo van der Pol, Carole Vuillerot, Alexander Burdeska, Muna El-Khairi, Paulo Fontoura, Jane Ives, Ksenija Gorni, Carol Reid, Sabine Fuerst-Recktenwald

Research output: Contribution to journalArticlepeer-review


In a previous Phase 2 study, olesoxime had a favorable safety profile. Although the primary endpoint was not met, analyses suggested that olesoxime might help in the maintenance of motor function in patients with Types 2/3 SMA. This open-label extension study (OLEOS) further characterizes the safety, tolerability and efficacy of olesoxime over longer therapy durations. In OLEOS, no new safety risks were identified. Compared to matched natural history data, patients treated with olesoxime demonstrated small, non-significant changes in motor function over 52 weeks. Motor function scores were stable for 52 weeks but declined over the remainder of the study. The greatest decline in motor function was seen in patients ≤15 years old, and those with Type 2 SMA had faster motor function decline versus those with Type 3 SMA. Previous treatment with olesoxime in the Phase 2 study was not protective of motor function in OLEOS. Respiratory outcomes were stable in patients with Type 3 SMA >15 years old but declined in patients with Type 2 SMA and in patients with Type 3 SMA ≤15 years old. Overall, with no stabilization of functional measures observed over 130 weeks, OLEOS did not support significant benefit of olesoxime in patients with SMA.

Original languageEnglish
Pages (from-to)959-969
Number of pages11
JournalNeuromuscular Disorders
Issue number12
Publication statusPublished - Dec 2020


  • Longitudinal study
  • Motor function measure
  • Natural history data
  • Olesoxime
  • Spinal muscular atrophy

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Neurology
  • Clinical Neurology
  • Genetics(clinical)


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