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  • 185 JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving risdiplam

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ABN Abstracts 2022
Poster presentations
185 JEWELFISH: Safety, pharmacodynamic and exploratory efficacy data in non-naïve patients with SMA receiving risdiplam
  1. Mariacristina Scoto1,
  2. Claudio Bruno2,
  3. Dirk Fischer3,
  4. Janbernd Kirschner4,
  5. Eugenio Mercuri5,
  6. Imogen Carruthers6,
  7. Marianne Gerber7,
  8. Heidemarie Kletzl7,
  9. Francis Warren6,
  10. Claudia Chiriboga,
  11. on behalf of the JEWELFISH Working Group8
  1. 1Great Ormond Street Hospital, London
  2. 2Istituto Giannina Gaslini, Genoa, Italy
  3. 3University Children’s Hospital Basel, Basel, Switzerland
  4. 4Medical Center-University of Freiburg, Freiburg, Germany
  5. 5Fondazione Policlinico Gemelli IRCCS, Rome, Italy
  6. 6Roche Products Ltd, UK
  7. 7F. Hoffmann-La Roche Ltd, Basel, Switzerland
  8. 8Columbia University Irving Medical Center, New York, NY, USA

Abstract

Risdiplam (EVRYSDI®) is an oral survival of motor neuron 2 (SMN2) pre-mRNA splicing modifier approved by the EMA and MHRA for the treatment of patients aged ≥2 months, with a clinical diagnosis of Type 1, 2 or 3 spinal muscular atrophy (SMA) or 1–4 SMN2 copies.

JEWELFISH (NCT03032172) is an open-label study of risdiplam in patients with SMA who previously received RG7800 (RO6885247), nusinersen (SPINRAZA®), olesoxime or onasemnogene abeparvovec (ZOLGENSMA®).

JEWELFISH assesses the safety, tolerability and pharmacokinetic/pharmacodynamic (PD) relationship of risdiplam in a broad range of ages (1–60 years), SMA types (1–3), SMN2 copy numbers (1–4) and motor function (nonsitters, sitters and walkers). We previously presented safety data from 173 patients (data cut-off: 31 July 2020): 13 previously received RG7800, 76 received nusinersen, 70 received olesoxime and 14 received onasemnogene abeparvovec. Risdiplam led to a rapid and sustained >2-fold increase in SMN protein (data cut-off: 1 June 2020), consistent with data from treatment-naïve patients with Types 2/3 SMA (SUNFISH; NCT02908685).

No drug-related safety findings leading to withdrawal were reported for any patients in JEWELFISH. The safety profile of risdiplam was consistent with observations in treatment-naïve patients. Here we present 12-month safety, PD and exploratory efficacy data.

https://doi.org/10.1136/jnnp-2022-abn2.229

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