Risdiplam
- Generic Name
- Risdiplam
- Brand Names
- Evrysdi
- Drug Type
- Small Molecule
- Chemical Formula
- C22H23N7O
- CAS Number
- 1825352-65-5
- Unique Ingredient Identifier
- 76RS4S2ET1
- Background
Risdiplam is an orally bioavailable mRNA splicing modifier used for the treatment of spinal muscular atrophy (SMA). It increases systemic SMN protein concentrations by improving the efficiency of SMN2 gene transcription. This mechanism of action is similar to its predecessor nusinersen, the biggest difference being their route of administration: nusinersen requires intrathecal administration, as does the one-time gene therapy onasemnogene abeparvovec, whereas risdiplam offers the ease of oral bioavailability.
Risdiplam was approved by the FDA in August 2020 for the treatment of spinal muscular atrophy (SMA). Set to be substantially cheaper than other available SMA therapies, risdiplam appears to provide a novel and relatively accessible treatment option for patients with SMA regardless of severity or type.
- Indication
Risdiplam is indicated for the treatment of spinal muscular atrophy (SMA).
- Associated Conditions
- Spinal Muscular Atrophy (SMA)
Adult SMA Research and Clinical Hub
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- First Posted Date
- 2025-05-18
- Last Posted Date
- 2025-05-18
- Lead Sponsor
- Newcastle-upon-Tyne Hospitals NHS Trust
- Target Recruit Count
- 600
- Registration Number
- NCT06978985
- Locations
- 🇬🇧
University Hospitals Birmingham NHS Foundation Trust, Birmingham, United Kingdom
🇬🇧North Bristol NHS Trust, Bristol, United Kingdom
🇬🇧Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
- First Posted Date
- 2023-05-17
- Last Posted Date
- 2025-05-13
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 28
- Registration Number
- NCT05861999
- Locations
- 🇺🇸
Children'S Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇺🇸University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
🇺🇸Valley Children's Hospital, Madera, California, United States
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
- First Posted Date
- 2023-05-17
- Last Posted Date
- 2025-05-18
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 28
- Registration Number
- NCT05861986
- Locations
- 🇵🇱
Instytut Pomnik Centrum Zdrowia Dziecka, Warszawa, Poland
🇺🇸University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
🇺🇸Children's Hospital of Colorado, Aurora, Colorado, United States
A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
- First Posted Date
- 2023-04-11
- Last Posted Date
- 2025-05-20
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 10
- Registration Number
- NCT05808764
- Locations
- 🇺🇸
University Of Michigan, Ann Arbor, Michigan, United States
🇵🇱Instytut Pomnik - Centrum Zdrowia Dziecka, Warszawa, Poland
🇺🇸Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
- First Posted Date
- 2022-08-31
- Last Posted Date
- 2024-02-07
- Lead Sponsor
- Clinic for Special Children
- Target Recruit Count
- 10
- Registration Number
- NCT05522361
- Locations
- 🇺🇸
Clinic for Special Children, Strasburg, Pennsylvania, United States
Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- Conditions
- Spinal Muscular Atrophy
- Interventions
- Device: SMA-DAT Application and ADAM Sensor
- First Posted Date
- 2022-02-10
- Last Posted Date
- 2025-03-06
- Lead Sponsor
- Genentech, Inc.
- Target Recruit Count
- 402
- Registration Number
- NCT05232929
- Locations
- 🇺🇸
Barrow Neurological Institute, Phoenix, Arizona, United States
🇺🇸Phoenix Children's Hospital, Phoenix, Arizona, United States
🇺🇸University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States
Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
- Conditions
- Spinal Muscular Atrophy
- First Posted Date
- 2022-02-02
- Last Posted Date
- 2024-12-31
- Lead Sponsor
- Bakri Elsheikh
- Target Recruit Count
- 30
- Registration Number
- NCT05219487
- Locations
- 🇺🇸
The Ohio State University Wexner Medical Center, Columbus, Ohio, United States
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
- Conditions
- Spinal Muscular Atrophy (SMA)
- Interventions
- First Posted Date
- 2021-11-10
- Last Posted Date
- 2025-03-24
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 259
- Registration Number
- NCT05115110
- Locations
- 🇺🇸
Nemours Children's Hospital, Orlando, Florida, United States
🇧🇪Chr de La Citadelle, Liège, Belgium
🇵🇹Hospital de Santa Maria, Lisboa, Portugal
Bioavailability and Bioequivalence of Two Risdiplam Tablets in Healthy Participants
- First Posted Date
- 2021-01-22
- Last Posted Date
- 2023-03-13
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 131
- Registration Number
- NCT04718181
- Locations
- 🇺🇸
Dallas Clinical Research Unit, Dallas, Texas, United States
🇺🇸Daytona Beach Clinical Rsch Unit, Daytona Beach, Florida, United States
🇺🇸Covance Clinical Research Unit, Inc, Madison, Wisconsin, United States
An Expanded Access Program for Risdiplam in Participants With Spinal Muscular Atrophy (SMA)
- Conditions
- Muscular Atrophy, Spinal
- First Posted Date
- 2020-02-05
- Last Posted Date
- 2020-10-05
- Lead Sponsor
- Genentech, Inc.
- Registration Number
- NCT04256265
- Locations
- 🇺🇸
Arkansas Children's Hospital; Pediatrics, Little Rock, Arkansas, United States
🇺🇸Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.