131  Pooled safety data from the risdiplam clinical trial development programme. Issue 6 (27th May 2022)

• Record Type: Journal Article
• Authors: Servais, Laurent; Baranello, Giovanni; Chiriboga, Claudia; Darras, Basil; Bader-Weder, Silvia; Gorni, Ksenija; Jaber, Birgit; McIver, Tammy; Scalco, Renata
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 93:Issue 6(2022)
• Page Start: A142
• Page End: A142
• Publication Date: 2022-05-27
• Subjects: Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2022-ABN.456
• Languages: English
• ISSNs: 0022-3050

Abstract

Abstract : Risdiplam is a centrally and peripherally distributed oral survival of motor neuron 2 (SMN2) pre mRNA splicing modifier that increases the levels of functional SMN protein. Risdiplam (EVRYSDI™) has been approved by the US Food and Drug Administration for the treatment of patients with spinal muscular atrophy (SMA), aged 2 months and older. The risdiplam clinical development programme consists of four studies in a broad population of individuals with SMA. FIREFISH (NCT02913482 ) and SUNFISH (NCT02908685 ) are two-part studies assessing safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD) and efficacy in infants with Type 1 SMA and patients with Type 2/3 SMA, respectively. JEWELFISH (NCT0302172) assesses safety, tolerability, PK and PD in patients with SMA who previously received RG7800 (R06885247), nusinersen (SPINRAZA®), olesoxime or onasemno- gene abeparvovec-xioi (ZOLGENSMA®). RAINBOWFISH (NCT03779334 ) assesses efficacy, safety, PK and PD in infants with genetically diagnosed and presymptomatic SMA. Pooled analyses of FIREFISH and SUNFISH Parts 1 and 2 and JEWELFISH were conducted to determine the long-term safety profile of risdiplam. At the data-cut (15th January 2020) no treatment-related safety findings led to withdrawal from up to 39 months' risdiplam treatment in 465 patients. Here we will present updated pooled safety analyses for the risdiplam studies. [e-mail]