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)
A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants
- First Posted Date
- 2019-06-18
- Last Posted Date
- 2020-10-19
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 35
- Registration Number
- NCT03988907
- Locations
- 🇺🇸
Covance Research Unit - Dallas, Dallas, Texas, United States
A Study to Investigate the Effect of Hepatic Impairment on the Pharmacokinetics and Safety and Tolerability of a Single Oral Dose of Risdiplam Compared to Matched Healthy Participants With Normal Hepatic Function
- First Posted Date
- 2019-04-19
- Last Posted Date
- 2021-02-21
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 26
- Registration Number
- NCT03920865
- Locations
- 🇺🇸
Clinical Pharmacology of Miami, Inc., Miami, Florida, United States
🇺🇸Orlando Clinical Research Center, Orlando, Florida, United States
🇺🇸American Research Corporation Inc., San Antonio, Texas, United States
A Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
- First Posted Date
- 2018-12-19
- Last Posted Date
- 2025-03-20
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 26
- Registration Number
- NCT03779334
- Locations
- 🇦🇺
Sydney Children's Hospital, Randwick, New South Wales, Australia
🇧🇷Hospital das Clinicas - FMUSP_X, Sao Paulo, São Paulo, Brazil
🇵🇱Szpital Gdanskiego Uniwersytetu Medycznego, Gda?sk, Poland
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) in Healthy Japanese Participants
- First Posted Date
- 2017-02-02
- Last Posted Date
- 2018-10-04
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 24
- Registration Number
- NCT03040635
- Locations
- 🇺🇸
Collaborative Neuroscience Network, Inc., Garden Grove, California, United States
A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy
- First Posted Date
- 2017-01-26
- Last Posted Date
- 2025-02-24
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 174
- Registration Number
- NCT03032172
- Locations
- 🇺🇸
Nemours Children's Hospital, Orlando, Florida, United States
🇺🇸Boston Childrens Hospital, Boston, Massachusetts, United States
🇺🇸Columbia University Medical Center, New York, New York, United States
Investigate Safety, Tolerability, PK, PD and Efficacy of Risdiplam (RO7034067) in Infants With Type1 Spinal Muscular Atrophy
- First Posted Date
- 2016-09-23
- Last Posted Date
- 2024-08-07
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 62
- Registration Number
- NCT02913482
- Locations
- 🇺🇸
Ann and Robert H. Lurie Children Hospital of Chicago, Chicago, Illinois, United States
🇵🇱Szpital Gdanskiego Uniwersytetu Medycznego; Clinic of developmental neurology, Gda?sk, Poland
🇺🇦Ams of Ukraine; Inst. of Neurology, Psychiatry & Narcology, Kharkov, Ukraine
A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants
- First Posted Date
- 2016-09-21
- Last Posted Date
- 2024-04-24
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 231
- Registration Number
- NCT02908685
- Locations
- 🇯🇵
Minami Kyushu National Hospital, Kagoshima, Japan
🇯🇵Miyagi Children's Hospital, Miyagi, Japan
🇯🇵Shiga Medical Center for Children, Shiga, Japan
A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers
- Conditions
- Spinal Muscular Atrophy
- Interventions
- First Posted Date
- 2015-12-17
- Last Posted Date
- 2018-10-04
- Lead Sponsor
- Hoffmann-La Roche
- Target Recruit Count
- 33
- Registration Number
- NCT02633709
- Locations
- 🇳🇱
Pra International Group B.V, Groningen, Netherlands
Drug Development Updates
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