Risdiplam

also known as Evrysdi · EvrysdiTM · RG-7916 · RO7034067

Developed for: spinal muscular atrophy
Investigated by: Hoffmann-La Roche · Genentech, Inc. · Clinic for Special Children

Regulatory milestones approvals, filings & regulatory actions · 5 recorded

Milestone	Jurisdiction	Brand	Indication	Date	Sentence it was read from
Label expansions (3)	US (FDA)	EVRYSDI	—	2022-05-27 – 2024-09-17	fda.gov ↗
Approved	EU (EMA)	Evrysdi	—	2021-03-26	europa.eu ↗
Approved	US (FDA)	EVRYSDI	—	2020-08-07	fda.gov ↗

Trials 15 · a red edge is where a trial was stopped

today

US approved 2020

EU approved 2021

NCT05861999 · Ph 4 NCT05861986 · Ph 4 NCT05808764 · Ph 2 NCT05522361 · Ph 4 NCT05232929 · Ph 4 NCT03779334 · Ph 2 NCT03988907 · Ph 1 NCT03920865 · Ph 1 NCT03040635 · Ph 1 NCT03032172 · Ph 2 NCT03036501 · Ph 1 NCT02913482 · Ph 2 NCT02908685 · Ph 2 NCT02633709 · Ph 1

2016201720182019202020212022202320242025202620272028

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 15 trials
Phase 1	NCT03988907	Jun → Sep 2019	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 1	NCT03920865	May 2019 → Jan 2020	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 1	NCT03040635	Mar → Oct 2017	—	Hoffmann-La Roche	Completed	No outcome recorded
Phase 1	NCT03036501	Jan → Feb 2017	—	Hoffmann-La Roche	Completed	No outcome recorded
Phase 1	NCT02633709	Jan → Aug 2016	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 25 trials
Phase 2	NCT05808764	Apr 2024 → May 2026	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 2	NCT03779334	Aug 2019 → Feb 2023 overdue	spinal muscular atrophy	Hoffmann-La Roche	Active not recruiting	No outcome recorded
Phase 2	NCT03032172	Mar 2017 → Feb 2025	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 2	NCT02913482	Dec 2016 → Nov 2019	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 2	NCT02908685	Oct 2016 → Sep 2019	spinal muscular atrophy	Hoffmann-La Roche	Completed	No outcome recorded
Phase 44 trials
Phase 4	NCT05861999	Aug 2024 → Mar 2028 expected	spinal muscular atrophy	Hoffmann-La Roche	Recruiting	No outcome recorded
Phase 4	NCT05861986	May 2024 → Mar 2028 expected	spinal muscular atrophy	Hoffmann-La Roche	Recruiting	No outcome recorded
Phase 4	NCT05522361	Nov 2022 → Dec 2025	spinal muscular atrophy	Clinic for Special Children	Unknown	No outcome recorded
Phase 4	NCT05232929	Mar 2022 → Dec 2026 expected	spinal muscular atrophy	Genentech, Inc.	Active not recruiting	No outcome recorded
Phase not stated1 trial
—	NCT04256265	—	spinal muscular atrophy	Genentech, Inc.	Approved for marketing	No outcome recorded

Also used as a comparator or background therapy in 2 trials

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 21 trial
Phase 2	NCT07047144 Background	Sep 2025 → Nov 2028 expected	spinal muscular atrophy, type II, spinal muscular atrophy, type III	Scholar Rock, Inc.	Recruiting	No outcome recorded
Phase 2/31 trial
Phase 2/3	NCT05115110 Background	Jun 2022 → Oct 2026 expected	spinal muscular atrophy	Hoffmann-La Roche	Active not recruiting	No outcome recorded

Press releases naming this drug 33 releases

Date	Issuer	Release
2025-06-04	Roche Global Development	Roche’s Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA) roche.com ↗
2025-02-12	Roche Global Development	FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA) roche.com ↗
2024-10-14	Roche Global Development	Majority of children with spinal muscular atrophy (SMA) treated with Roche’s Evrysdi are able to sit, stand and walk independently, two-year data demonstrate roche.com ↗
2024-06-07	Roche Global Development	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 roche.com ↗
2023-10-04	Roche Global Development	Majority of newborn babies with spinal muscular atrophy (SMA) treated with Roche’s Evrysdi able to sit independently after 1 year of treatment roche.com ↗
2023-08-29	Roche Global Development	European Commission approves Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA) roche.com ↗
2023-07-21	Roche Global Development	CHMP recommends Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA) roche.com ↗
2023-06-30	Roche Global Development	Four-year follow up data for Roche’s Evrysdi show continued increase in number of children with a severe form of spinal muscular atrophy (SMA) able to sit, stand and walk roche.com ↗
2023-03-20	Roche Global Development	New four year data for Roche’s Evrysdi reinforce long-term efficacy and safety profile in some of the most severely affected people with types 2 and 3 spinal muscular atrophy (SMA) roche.com ↗
2022-10-12	Roche Global Development	Positive new data for Roche’s Evrysdi in largest trial ever undertaken in patients with previously-treated spinal muscular atrophy (SMA) roche.com ↗
2022-05-31	Roche Global Development	FDA approves Roche’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA) roche.com ↗
2022-04-29	Roche Global Development	New three-year data for Roche’s Evrysdi (risdiplam) show long-term improvements in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA) roche.com ↗
2022-03-16	Roche Global Development	New data for Roche’s Evrysdi (risdiplam) demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy (SMA) roche.com ↗
2022-03-08	Roche Global Development	Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio roche.com ↗
2022-01-25	Roche Global Development	Roche’s Evrysdi (risdiplam) granted FDA priority review for treatment of pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA) roche.com ↗
2021-07-29	Roche Global Development	Data for Roche’s Evrysdi® (risdiplam) published in New England Journal of Medicine shows significant improvement in survival and motor milestones in babies with Type 1 spinal muscular atrophy (SMA) roche.com ↗
2021-06-11	Roche Global Development	New Roche data for Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with spinal muscular atrophy (SMA) roche.com ↗
2021-04-15	Roche Global Development	Roche’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA) roche.com ↗
2021-03-30	Roche Global Development	Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy roche.com ↗
2021-03-16	Roche Global Development	New two-year data show Roche’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA) roche.com ↗
2021-02-26	Roche Global Development	Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older roche.com ↗
2021-02-25	Roche Global Development	Roche announces results from Evrysdi (risdiplam) study in infants with Type 1 spinal muscular atrophy (SMA) published in New England Journal of Medicine roche.com ↗
2020-09-28	Roche Global Development	Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA) roche.com ↗
2020-08-10	Roche Global Development	FDA approves Roche’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older roche.com ↗
2020-06-12	Roche Global Development	Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA) roche.com ↗
2020-04-28	Roche Global Development	Roche’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA) roche.com ↗
2020-04-07	Roche Global Development	Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA) roche.com ↗
2020-02-06	Roche Global Development	Roche’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy roche.com ↗
2020-01-23	Roche Global Development	Roche’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy roche.com ↗
2019-11-25	Roche Global Development	FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy roche.com ↗
2019-11-11	Roche Global Development	Roche’s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy roche.com ↗
2019-05-07	Roche Global Development	Roche presents data from the risdiplam pivotal FIREFISH and SUNFISH studies in spinal muscular atrophy at the 2019 AAN Annual Meeting roche.com ↗
2018-12-17	Roche Global Development	PRIME designation granted by European Medicines Agency for Roche's risdiplam for treatment of spinal muscular atrophy (SMA) roche.com ↗

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 20 sources stand behind the page.

Field	Value	Cited text
Known as	Risdiplam	ClinicalTrials.gov intervention name — accepted as the source's own label NCT05861999 ↗ 16 NCT05861986 ↗ NCT03779334 ↗ NCT05808764 ↗ NCT02908685 ↗ NCT03036501 ↗ NCT03040635 ↗ NCT02913482 ↗ NCT05522361 ↗ NCT05232929 ↗ NCT05115110 ↗ NCT02633709 ↗ NCT03032172 ↗ NCT03920865 ↗ NCT07047144 ↗ NCT03988907 ↗ NCT04256265 ↗
Known as	Evrysdi	ChEMBL registry synonym — accepted as the source's own label CHEMBL4297528 ↗
Known as	EvrysdiTM	“Risdiplam (EvrysdiTM) increases SMN protein and is approved for the treatment of SMA.” PMID 37148485 ↗ May 2023
Known as	RG-7916	ChEMBL registry synonym — accepted as the source's own label CHEMBL4297528 ↗
Known as	RO7034067	“labeled Risdiplam (RO7034067)” NCT03036501 ↗
Action	Restore	“increases tissue SMN protein levels” NCT05522361 ↗
Modality	Small molecule	“Risdiplam is an oral small molecule approved for the treatment of patients with spinal muscular atrophy” PMID 34942136 ↗ Jan 2022
Route	Oral	“Risdiplam (RO7034067) will be given by mouth” NCT02633709 ↗
Target	SMN2	“The drug modifies pre-mRNA splicing of the SMN2 gene to increase production of functional SMN.” PMID 34942136 ↗ Jan 2022