Evrysdi (risdiplam) has been added to the MAF List to improve access to SMA treatment for children and adults with Types 1, 2 and 3 SMA across four patient groups; it is the first treatment for SMA to be included on the MAF List.1
The Ministry of Health Drug Advisory Committee recommended Evrysdi for subsidy under the MAF Subsidy Framework, alongside additional clinical criteria developed in consultation with local clinical experts from public healthcare institutions.1
Evrysdi has demonstrated efficacy in adults, children and infants 16 days and older across four pivotal clinical trials; it is now approved in more than 100 countries with over 15,000 patients treated globally.2-8
SINGAPORE, 01 August, 2024 – EVRYSDI® (risdiplam) has been added to the Singapore Ministry of Health’s (MOH) list of medicines covered by the Medication Assistance Fund (MAF) Subsidy Framework, making it the first treatment for spinal muscular atrophy (SMA) to be included. Under the MAF Subsidy Framework, patients with SMA, who are either Singaporeans or Permanent Residents and meet the clinical criteria, are eligible for subsidised treatment with Evrysdi in public healthcare institutions.
MAF subsidies for Evrysdi will improve access to the drug for SMA treatment in four patient populations:
● Children with symptomatic Type 1, 2 or 3 SMA
● Adults with symptomatic Type 1, 2 or 3 SMA;
● Children with pre-symptomatic SMA; and
● Patients with SMA who have experienced a regression in a developmental state despite treatment with gene therapy
SMA is a rare neuromuscular disorder that causes muscle weakness and wasting, leading to symptoms such as breathing difficulties and loss of motor function. In Singapore, 40 to 60 patients have been diagnosed with SMA. In addition, there are about three babies born each year with SMA.1 The most severe form is Type 1 SMA, which affects about 60% of patients and typically presents within the first six months of life.8 Untreated, most children with Type 1 SMA do not survive past their second birthday.10-11
In the FIREFISH clinical trial, 58 children aged 7 months or younger with Type 1 SMA were treated with Evrysdi.6 After five years, 91% remained alive, 96% could swallow, 80% could feed without a feeding tube, and 59% could sit unsupported for at least 30 seconds.12 In the SUNFISH Part 2 trial, children and adults with Type 2 or 3 SMA who received Evrysdi over 12 months showed improvements in motor function scores that were maintained over the long-term.3-4 Two other trials, JEWELFISH and RAINBOWFISH, also demonstrated Evrysdi’s efficacy in paediatric and adult patients who had received prior SMA-targeted therapies, and presymptomatic newborns diagnosed with SMA, respectively.7-8
Ying Ying Yeoh, General Manager, Roche Pharmaceuticals, Singapore, said “It is our honour to contribute in this effort to improve access to Evrysdi for patients with SMA in Singapore. Roche remains committed to constantly innovating to improve the lives of people with neurological and neuromuscular disorders.”
Judy Wee, Executive Director, Muscular Dystrophy Association (Singapore), said “The inclusion of Evrysdi on MAF today is a milestone that will have impact far beyond treatment of the disease. With more affordable and effective treatment, patients with SMA have the potential to retain or reclaim their independence, while alleviating some of the burden placed on caregivers and loved ones.”
Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth.
Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the CNS and peripheral tissues. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions such as swallowing, speaking, and breathing.
Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research Award for Drug Discovery. Evrysdi is currently approved in more than 100 countries, and the dossier is under review in a further 13 countries.
Evrysdi is currently being, or has been, evaluated in five global multicentre trials in people with SMA:
● FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study.The study met its primary endpoint.
● SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. The study met its primary endpoint.
● JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174).
● RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicentre study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint.
● MANATEE (NCT05115110) – a phase 2/3 clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is currently recruiting.
SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.
Neuroscience is a major focus of research and development at Roche. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases.
Roche is investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today.
References
Ministry of Health Drug Advisory Committee (2024) Technology Guidance: Nusinersen and risdiplam for treating spinal muscular atrophy. Available at
Evrysdi Singapore Prescribing Information. June 2023. Available at
Mercuri E, Deconinck N, Mazzone ES, et al. Safety and efficacy of once-daily risdiplam in type 2 and non-ambulant type 3 spinal muscular atrophy (SUNFISH part 2): a phase 3, double-blind, randomised, placebo-controlled trial [published correction appears in Lancet Neurol. 2022 Feb;21(2):e2. doi: 10.1016/S1474-4422(22)00006-0] [published correction appears in Lancet Neurol. 2022 Mar;21(3):e3. doi: 10.1016/S1474-4422(22)00038-2] [published correction appears in Lancet Neurol. 2022 May;21(5):e5. doi: 10.1016/S1474-4422(22)00141-7]. Lancet Neurol. 2022;21(1):42-52. doi:10.1016/S1474-4422(21)00367-7
Oskoui M, Day JW, Deconinck N, et al. Two-year efficacy and safety of risdiplam in patients with type 2 or non-ambulant type 3 spinal muscular atrophy (SMA) [published correction appears in J Neurol. 2023 May;270(5):2547-2549. doi: 10.1007/s00415-023-11658-6]. J Neurol. 2023;270(5):2531-2546. doi:10.1007/s00415-023-11560-1
Day, J. et al. (2022) ‘P.114 Sunfish Parts 1 and 2: 3-year efficacy and safety of risdiplam in types 2 and 3 spinal muscular atrophy (SMA)’, Neuromuscular Disorders, 32. doi:10.1016/j.nmd.2022.07.199.
Masson R, Mazurkiewicz-Bełdzińska M, Rose K, et al. Safety and efficacy of risdiplam in patients with type 1 spinal muscular atrophy (FIREFISH part 2): secondary analyses from an open-label trial. Lancet Neurol. 2022;21(12):1110-1119. doi:10.1016/S1474-4422(22)00339-8.
Chiriboga CA, Bruno C, Duong T, et al. Risdiplam in Patients Previously Treated with Other Therapies for Spinal Muscular Atrophy: An Interim Analysis from the JEWELFISH Study [published correction appears in Neurol Ther. 2023 Oct;12(5):1799-1801. doi: 10.1007/s40120-023-00503-7]. Neurol Ther. 2023;12(2):543-557. doi:10.1007/s40120-023-00444-1
Finkel, R. et al. (2022) ‘FP.24 rainbowfish: Preliminary efficacy and safety data in RISDIPLAM-treated infants with presymptomatic spinal muscular atrophy (SMA)’, Neuromuscular Disorders, 32. doi:10.1016/j.nmd.2022.07.183.
Verhaart IEC, Robertson A, Wilson IJ, Aartsma-Rus A, Cameron S, Jones CC, Cook SF, Lochmüller H. Prevalence, incidence and carrier frequency of 5q-linked spinal muscular atrophy - a literature review. Orphanet J Rare Dis. 2017 Jul 4;12(1):124. doi: 10.1186/s13023-017-0671-8. PMID: 28676062; PMCID: PMC5496354.
Spinal muscular atrophy (SMA), Cleveland Clinic. Available at:
Finkel S, et al. Observational study of spinal muscular atrophy type I and implications for clinical trials. American Academy of Neurology. 2014; 83:811.
‘Five-year data for Roche’s Evrysdi show the majority of treated children with a severe form of spinal muscular atrophy (SMA) achieved or maintained the ability to sit, stand or walk’ (2024) Roche.com [Preprint]. Available at: https://www.roche.com/media/releases/med-cor-2024-06-07 (Accessed: 26 June 2024)
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