Drugs / Risdiplam
Regulatory milestones approvals, filings & regulatory actions · 7 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed arthrogryposis spinal muscular atrophy 2 milestones | |||||
| Filed | EU (EMA) | Evrysdi | arthrogryposis spinal muscular atrophy | 2020-08-17 | “today announced the acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for Evrysdi™ (risdiplam)” ptcbio.com ↗ |
| Filed | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2019-11-24 | “today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam.” gene.com ↗ “today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam” gene.com ↗ “In November 2019, the U.S. Food and Drug Administration (FDA) accepted a New Drug Application (NDA) and granted Priority Review for risdiplam.” ptcbio.com ↗ |
| Approved Indication not stated 3 milestones | |||||
| 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
| 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 · 3 met primary | ||||||
| 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 | Met primary |
| 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 | Met primary |
| Phase 2 | NCT02908685 | Oct 2016 → Sep 2019 | spinal muscular atrophy | Hoffmann-La Roche | Completed | Met primary |
| 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 → Oct 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 |
| Comparator or background therapy2 trials | ||||||
| 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 2/3 | NCT05115110 Background | Jun 2022 → Oct 2026 expected | spinal muscular atrophy | Hoffmann-La Roche | Active not recruiting | Met primary |
News releases announcing trial results or a regulatory action · 92
| Date | Issuer | Release |
|---|---|---|
| 2025-06-04 | Hoffmann-La Roche | Regulatory Roche’s Evrysdi tablet approved by European Commission as first and only for Spinal Muscular Atrophy (SMA) roche.com ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission (EC) has approved a label extension for Evrysdi® (risdiplam) to include a new, room-temperature stable tablet for people living with spinal muscular atrophy (SMA). |
| 2025-03-17 | Hoffmann-La Roche | Results Roche to share latest scientific advancements from its neuromuscular portfolio at Muscular Dystrophy Association (MDA) 2025 conference roche.com ↗
New Evrysdi five-year data from the SUNFISH study showed continued stabilisation of motor function in a broad population of individuals with Types 2 or 3 spinal muscular atrophy (SMA) |
| 2025-02-12 | Hoffmann-La Roche | Regulatory FDA approves Roche’s Evrysdi tablet as first and only tablet for Spinal Muscular Atrophy (SMA) roche.com ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). |
| 2025-02-12 | Genentech, Inc. | Regulatory FDA Approves Genentech’s Evrysdi Tablet as First and Only Tablet for Spinal Muscular Atrophy (SMA) gene.com ↗
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved a New Drug Application (NDA) for an Evrysdi® (risdiplam) tablet for people living with spinal muscular atrophy (SMA). |
| 2024-10-14 | Hoffmann-La Roche | Results 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 ↗
Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before six weeks of age, with most achieving motor milestones similar to children without SMA |
| 2024-10-13 | Genentech, Inc. | Results Majority of Children With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Are Able to Sit, Stand and Walk Independently, Two-Year Data Demonstrate gene.com ↗
The study found the majority of children achieved key motor milestones, were able to swallow and feed orally, and demonstrated cognitive skills typical of children without SMA, with none requiring permanent ventilation. |
| 2024-09-24 | Chugai Pharmaceutical | Regulatory Chugai Receives Approvals for Additional Indication of Evrysdi in Pre-Symptomatic Spinal Muscular Atrophy (SMA) and Additional Dosage for SMA Infants Under 2 Months of Age chugai-pharm.co.jp ↗
We are very pleased that with this approval, we can now deliver Evrysdi to people with SMA of all ages after birth, regardless of symptom onset. |
| 2024-06-07 | Hoffmann-La Roche | Results 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 ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today new five-year data confirming the sustained efficacy and safety profile of Evrysdi® (risdiplam) in children with Type 1 spinal muscular atrophy (SMA) from the open-label extension of the pivotal FIREFISH study. |
| 2024-06-07 | Genentech, Inc. | Results Five-Year Data for Genentech’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 gene.com ↗
By the end of year 5, 91% of children treated with Evrysdi were alive, 81% were alive without permanent ventilation and the majority were able to sit without support for at least 30 seconds (59%). |
| 2023-10-04 | Hoffmann-La Roche | Results 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 ↗
The study met its primary endpoint with 80% of the primary efficacy population (n=5) sitting without support for at least five seconds after 1 year of Evrysdi treatment, assessed by Bayley Scales of Infant and Toddler Development, third edition (BSID-III). |
| 2023-10-03 | Genentech, Inc. | Results Majority of Newborn Babies With Spinal Muscular Atrophy (SMA) Treated With Genentech’s Evrysdi Able to Sit Independently After 1 Year of Treatment gene.com ↗
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented positive results from the primary analysis of the ongoing RAINBOWFISH study assessing the efficacy and safety of Evrysdi® (risdiplam) in babies with pre-symptomatic SMA (n=26), aged from birth to 6 weeks. |
| 2023-08-29 | Hoffmann-La Roche | Regulatory European Commission approves Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA) roche.com ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today that the European Commission has approved the extension of the Evrysdi® (risdiplam) European Union (EU) marketing authorisation to include infants with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies from birth to below two months. |
| 2023-07-21 | Hoffmann-La Roche | Regulatory CHMP recommends Roche’s Evrysdi for babies under two months old with spinal muscular atrophy (SMA) roche.com ↗ |
| 2023-07-21 | PTC Therapeutics | Regulatory CHMP Recommends Evrysdi® for Babies Under Two Months Old with Spinal Muscular Atrophy ptcbio.com ↗ |
| 2023-06-30 | Hoffmann-La Roche | Results 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 ↗
After four years of treatment with Evrysdi, many of the babies, now young children, continued to improve their ability to sit, stand and walk without support. |
| 2023-06-30 | Genentech, Inc. | Results Four-Year Follow-Up Data for Genentech’s Evrysdi Show Continued Increase in Number of Children With a Severe Form of Spinal Muscular Atrophy (SMA) Able to Sit, Stand and Walk gene.com ↗
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new long-term data for Evrysdi® (risdiplam) from the open-label extension (n=50) of the pivotal FIREFISH study, reinforcing its sustained efficacy and safety profile in children with Type 1 spinal muscular atrophy (SMA). |
| 2023-04-24 | Genentech, Inc. | Results Genentech Announces Industry-Leading Brain Health Research Collaborations and Latest Data Across Neuroscience Medicines at AAN 2023 Annual Meeting gene.com ↗
Long-term data to be presented demonstrate delayed MS disability with Ocrevus; reduced disease activity with Enspryng in NMOSD and with Evrysdi in SMA |
| 2023-03-20 | Hoffmann-La Roche | Results 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 ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new long-term data for Evrysdi® (risdiplam) in a broad range of people aged 2-25 years with spinal muscular atrophy (SMA) from the pivotal SUNFISH study1. |
| 2023-03-19 | Genentech, Inc. | Results New Four-Year Data for Genentech’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) gene.com ↗
Data confirm increases in motor function were sustained at four years and the overall rate of adverse events continued to decrease over the 48-month period, reinforcing the long-term efficacy and safety of Evrysdi. |
| 2022-10-12 | Hoffmann-La Roche | Results Positive new data for Roche’s Evrysdi in largest trial ever undertaken in patients with previously-treated spinal muscular atrophy (SMA) roche.com ↗
Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after 2-years of treatment. |
| 2022-10-11 | Genentech, Inc. | Results Positive New Data for Genentech’s Evrysdi in Largest Trial Ever Undertaken in Patients With Previously-Treated Spinal Muscular Atrophy (SMA) gene.com ↗
Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after two years of treatment. |
| 2022-10-05 | Hoffmann-La Roche | Results Roche to present new data from its expanding neuromuscular disease portfolio at World Muscle Society 2022 roche.com ↗
Overall, babies maintained or improved their motor skills in terms of developmental milestones and motor function between Month 24 and Month 36. |
| 2022-05-31 | Hoffmann-La Roche | Regulatory FDA approves Roche’s Evrysdi for use in babies under two months with spinal muscular atrophy (SMA) roche.com ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include babies under two months old with spinal muscular atrophy (SMA). |
| 2022-05-31 | PTC Therapeutics | Regulatory FDA Approves Label Extension for Evrysdi® for Infants with Spinal Muscular Atrophy Under 2 Months Old ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved a label extension for Evrysdi® (risdiplam) to include infants under 2 months old with spinal muscular atrophy (SMA). |
| 2022-05-30 | Genentech, Inc. | Regulatory FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy (SMA) gene.com ↗
FDA Approves Genentech’s Evrysdi (risdiplam) For Use in Babies Under Two Months with Spinal Muscular Atrophy (SMA) |
| 2022-04-29 | Hoffmann-La Roche | Results 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 ↗
The data showed an estimated 91% of infants (n=58) treated with Evrysdi were alive after three years of treatment. |
| 2022-04-28 | Genentech, Inc. | Results New Three-Year Data for Genentech’s Evrysdi (risdiplam) Show Long-Term Improvements in Survival and Motor Milestones in Babies With Type 1 Spinal Muscular Atrophy (SMA) gene.com ↗
The data showed an estimated 91% of infants (n=58) treated with Evrysdi were alive after three years of treatment. |
| 2022-03-25 | Hoffmann-La Roche | Results Roche to present data across broad and impactful neuroscience portfolio at 2022 AAN Annual Meeting roche.com ↗
Evrysdi data continue to demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy |
| 2022-03-24 | Genentech, Inc. | Results Genentech to Present Data Across Broad and Impactful Neuroscience Portfolio at 2022 AAN Annual Meeting gene.com ↗
Evrysdi (risdiplam) data continue to demonstrate long-term efficacy and safety in a broad population of people with spinal muscular atrophy |
| 2022-03-16 | Hoffmann-La Roche | Results 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 ↗
Presentations included new three-year data from the SUNFISH study which further confirmed the long-term efficacy and safety of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA. |
| 2022-03-15 | Genentech, Inc. | Results New Data for Genentech’s Evrysdi (risdiplam) Demonstrate Long-Term Efficacy and Safety in a Broad Population of People With Spinal Muscular Atrophy (SMA) gene.com ↗
Presentations included new three-year data from the SUNFISH study which further confirmed the long-term efficacy and safety of Evrysdi in a broad population of people aged 2-25 years with Type 2 or Type 3 SMA. |
| 2022-03-08 | Hoffmann-La Roche | Results Roche to present new Evrysdi data at MDA 2022 and highlight expanding neuromuscular disease portfolio roche.com ↗
Latest interim results from the RAINBOWFISH study demonstrate that the majority of babies treated with Evrysdi for at least 12 months were able to sit, stand and walk within timeframes typical of healthy babies |
| 2022-01-25 | Hoffmann-La Roche | Regulatory 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 ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). |
| 2022-01-25 | PTC Therapeutics | Regulatory FDA Grants Evrysdi® Priority Review Based on Results From Treating Pre-Symptomatic Infants with Spinal Muscular Atrophy ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for Evrysdi® (risdiplam) to expand the indication to include pre-symptomatic infants under 2 months old with spinal muscular atrophy (SMA). |
| 2022-01-24 | Genentech, Inc. | Regulatory Genentech’s Evrysdi (risdiplam) Granted FDA Priority Review for Treatment of Pre-Symptomatic Babies Under 2 Months of Age With Spinal Muscular Atrophy (SMA) gene.com ↗
today announced that the U.S. Food and Drug Administration (FDA) has granted priority review of a supplemental new drug application (sNDA) for the use of Evrysdi® (risdiplam) to treat pre-symptomatic babies under two months of age with spinal muscular atrophy (SMA). |
| 2021-09-24 | Hoffmann-La Roche | Results Roche presents new data at World Muscle Society (WMS) 2021 highlighting new advances for people living with rare neuromuscular disorders roche.com ↗
New data show pre-symptomatic babies with spinal muscular atrophy (SMA) treated with Evrysdi maintained the ability to swallow |
| 2021-07-29 | Hoffmann-La Roche | Results 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 ↗
The study met its primary endpoint with 29% of infants (12/41) sitting without support for at least five seconds* by month 12, a milestone not seen in the natural course of the disease. |
| 2021-07-28 | Genentech, Inc. | Results Data for Genentech’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) gene.com ↗
The study met its primary endpoint with 29% of infants (12/41) sitting without support for at least 5 seconds* by month 12, a milestone not seen in the natural course of the disease. |
| 2021-07-28 | PTC Therapeutics | Results Significant Improvements in Survival and Developmental Milestones Demonstrated in Type 1 SMA Patients Treated with Evrysdi™ ptcbio.com ↗
This trial showed that symptomatic Type 1 spinal muscular atrophy (SMA) infants treated with Evrysdi™ (risdiplam) showed significant improvements in survival, motor milestones, and motor function compared to historical controls. |
| 2021-06-23 | PTC Therapeutics | Regulatory Evrysdi™ Approved in Japan for the Treatment of Spinal Muscular Atrophy ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Evrysdi™ (risdiplam) was approved in Japan by the Japanese Ministry of Health, Labor and Welfare for the treatment of spinal muscular atrophy (SMA). |
| 2021-06-15 | Hoffmann-La Roche | Results Roche data at EAN 2021 showcase significant impact of therapies across diverse neuroscience portfolio roche.com ↗
Data for EVRYSDI reinforce safety profile and efficacy in a broad spinal muscular atrophy (SMA) population, following recent EU approval |
| 2021-06-11 | Hoffmann-La Roche | Results 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 ↗
Interim exploratory efficacy data from JEWELFISH also suggest stabilization in motor function at one year of treatment as measured by change from baseline in motor function measure (MFM 32). |
| 2021-06-11 | PTC Therapeutics | Results Pre-symptomatic Infants with Spinal Muscular Atrophy Achieved Same Motor Milestones as Healthy Children After Treatment with Evrysdi™ in RAINBOWFISH ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced positive interim results from the ongoing RAINBOWFISH trial that demonstrated pre-symptomatic infants with spinal muscular atrophy (SMA) treated with Evrysdi were able to reach motor milestones such as sitting without support, rolling, crawling, standing unaided and walking independently. |
| 2021-06-10 | Genentech, Inc. | Results New Genentech Data for Evrysdi (risdiplam) Show Improved Motor Function in Pre-Symptomatic Babies After One Year and Confirm Safety Profile in Previously Treated People With Spinal Muscular Atrophy (SMA) gene.com ↗
Interim exploratory efficacy data from JEWELFISH also suggest stabilization in motor function at one year of treatment as measured by change from baseline in motor function measure (MFM-32). |
| 2021-04-15 | Hoffmann-La Roche | Results Roche’s Evrysdi continues to improve motor function and survival in babies with Type 1 Spinal Muscular Atrophy (SMA) roche.com ↗
The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. |
| 2021-04-15 | PTC Therapeutics | Results Continued Improvements in Motor Milestones in Infants with Type 1 Spinal Muscular Atrophy After Two Years of Treatment with Evrysdi™ ptcbio.com ↗
Data from Part 2 of the FIREFISH trial demonstrated that at 24 months of treatment, 93% (38/41) of infants were alive and 83% (34/41) were alive without permanent ventilation. |
| 2021-04-14 | Genentech, Inc. | Results Genentech’s Evrysdi Continues to Improve Motor Function and Survival in Babies With Type 1 Spinal Muscular Atrophy (SMA) gene.com ↗
The data showed Evrysdi continued to improve motor function between months 12 and 24, including the ability to sit without support. |
| 2021-04-08 | Hoffmann-La Roche | Results New Roche data at 2021 AAN highlight impact and breadth of expanding neuroscience portfolio roche.com ↗
EVRYSDI 2-year FIREFISH Part 2 data show improvement in motor function in infants with Type 1 spinal muscular atrophy (SMA) |
| 2021-04-07 | Genentech, Inc. | Results New Genentech Data at 2021 AAN Highlight Impact and Breadth of Expanding Neuroscience Portfolio gene.com ↗
Evrysdi (risdiplam) 2-year FIREFISH Part 2 data show improvement in motor function in infants with Type 1 spinal muscular atrophy (SMA) |
| 2021-03-30 | Hoffmann-La Roche | Regulatory Roche’s Evrysdi approved by European Commission as first and only at home treatment for spinal muscular atrophy roche.com ↗
Today’s approval of Evrysdi, the first and only SMA treatment with proven efficacy that can be taken at home, potentially transforms treatment options for a broad range of people with SMA living in the EU, |
| 2021-03-30 | PTC Therapeutics | Regulatory PTC Therapeutics Announces the European Approval of Evrysdi™ for the Treatment of Spinal Muscular Atrophy ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the European Commission (EC) has granted marketing authorization to Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in patients 2 months and older. |
| 2021-03-16 | Hoffmann-La Roche | Results 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 ↗
The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. |
| 2021-03-16 | PTC Therapeutics | Results Results from the Second Year of Evrysdi™ (risdiplam) Treatment Demonstrated Sustained Improvement of Motor Function in a Broad Range of SMA Patients ptcbio.com ↗
These results demonstrated that Evrysdi patients sustained or improved in motor function after 24 months of treatment. |
| 2021-03-15 | Genentech, Inc. | Results New 2-Year Data Show Genentech’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) gene.com ↗
The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. |
| 2021-02-26 | Hoffmann-La Roche | Regulatory 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 ↗
The CHMP recommendation was completed under the accelerated assessment pathway, which is offered to medicines deemed to be of major interest for public health and therapeutic innovation. |
| 2021-02-26 | PTC Therapeutics | Regulatory CHMP Adopts Positive Opinion for Evrysdi™ for the Treatment of Spinal Muscular Atrophy in Adults and Children Aged Two Months and Older ptcbio.com ↗
"Today's CHMP opinion marks another important advancement in ensuring Evrysdi is available to SMA patients around the globe," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. |
| 2021-02-25 | Hoffmann-La Roche | Results 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 ↗
The study also found that treatment with Evrysdi increased the levels of survival of motor neuron (SMN) protein by a median 1.9-fold from baseline in the high-dose cohort at 12 months. |
| 2021-02-24 | Genentech, Inc. | Results Genentech Announces Results From Evrysdi (risdiplam) Study in Infants With Type 1 Spinal Muscular Atrophy (SMA) Published in New England Journal of Medicine gene.com ↗
The study also found that treatment with Evrysdi increased the levels of survival of motor neuron (SMN) protein by a median 1.9-fold from baseline in the high-dose cohort at 12 months. |
| 2021-02-24 | PTC Therapeutics | Results Positive Data from FIREFISH Study of Evrysdi™ (risdiplam) in Infants with Type 1 Spinal Muscular Atrophy Published in The New England Journal of Medicine ptcbio.com ↗
Data from the study also demonstrated increased levels of survival motor neuron (SMN) protein, which is critical for maintaining nerve and muscle function. |
| 2020-10-21 | PTC Therapeutics | Regulatory Evrysdi™ (risdiplam) Continues to Make Global Progress for the Treatment of Spinal Muscular Atrophy ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that Evrysdi™ (risdiplam) was approved in Brazil by the National Health Surveillance Agency (ANVISA) for the treatment of spinal muscular atrophy (SMA). |
| 2020-09-28 | Hoffmann-La Roche | Results Roche presents new 2-year data for Evrysdi (risdiplam) in infants with Type 1 spinal muscular atrophy (SMA) roche.com ↗
This exploratory analysis showed that an estimated 88% of infants were alive and required no permanent ventilation at two years. |
| 2020-09-28 | PTC Therapeutics | Results Two-Year Data of Evrysdi™ (risdiplam) in Infants with Spinal Muscular Atrophy Demonstrate Continued Improvement of Developmental Milestones ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that two-year data from Part 1 of the FIREFISH study demonstrated that infants on Evrysdi™ (risdiplam) continued to improve and achieve motor milestones. |
| 2020-09-27 | Genentech, Inc. | Results Genentech Presents New 2-Year Data for Evrysdi (risdiplam) in Infants With Type 1 Spinal Muscular Atrophy (SMA) gene.com ↗
The 2-year results in infants treated with the therapeutic dose of Evrysdi (17/21) showed that they continued to improve and achieve motor milestones. |
| 2020-08-17 | PTC Therapeutics | Regulatory PTC Announces the Acceptance of the European Marketing Authorization Application for Evrysdi™ (risdiplam) for the Treatment of Spinal Muscular Atrophy ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the acceptance of the Marketing Authorization Application (MAA) by the European Medicines Agency (EMA) for Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA). |
| 2020-08-10 | Hoffmann-La Roche | Regulatory FDA approves Roche’s Evrysdi (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older roche.com ↗
Roche announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. |
| 2020-08-07 | Genentech, Inc. | Regulatory FDA Approves Genentech’s Evrysdi (risdiplam) for Treatment of Spinal Muscular Atrophy (SMA) in Adults and Children 2 Months and Older gene.com ↗
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam) for treatment of spinal muscular atrophy (SMA) in adults and children 2 months of age and older. |
| 2020-08-07 | PTC Therapeutics | Regulatory PTC Therapeutics Announces FDA Approval of Evrysdi™ (risdiplam) for the Treatment of Spinal Muscular Atrophy in Adults and Children 2 Months and Older ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has approved Evrysdi™ (risdiplam), the first at-home, orally administered treatment for spinal muscular atrophy (SMA) in adults and children 2 months and older. |
| 2020-06-12 | Hoffmann-La Roche | Results 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 ↗
The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. |
| 2020-06-12 | Genentech, Inc. | Results Genentech Announces 2-Year Risdiplam Data From SUNFISH and New Data From JEWELFISH in Infants, Children and Adults With Spinal Muscular Atrophy (SMA) gene.com ↗
The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. |
| 2020-06-12 | PTC Therapeutics | Results PTC Therapeutics Announces 2-year Data from Part 1 of SUNFISH and New Data from JEWELFISH Trials for Risdiplam in Patients with Spinal Muscular Atrophy ptcbio.com ↗
The results of an exploratory efficacy analysis from SUNFISH showed risdiplam significantly improved motor function after 24 months of treatment compared to natural history data (Motor Function Measure Scale (MFM) 3.99 point difference; p< 0.0001). |
| 2020-04-28 | Hoffmann-La Roche | Results Roche’s risdiplam shows significant improvement in survival and motor milestones in infants with Type 1 spinal muscular atrophy (SMA) roche.com ↗
FIREFISH Part 2 study met its primary endpoint by demonstrating a significant increase in motor milestones in infants aged 1-7 months after 12 months of treatment |
| 2020-04-28 | PTC Therapeutics | Results Confirmatory Part 2 of FIREFISH Demonstrates Survival and Motor Milestones Not Seen in Natural History in Infants with Type 1 Spinal Muscular Atrophy ptcbio.com ↗
The global study met its primary endpoint of infants sitting without support for five seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). |
| 2020-04-27 | Genentech, Inc. | Results Genentech’s Risdiplam Shows Significant Improvement in Survival and Motor Milestones in Infants With Type 1 Spinal Muscular Atrophy (SMA) gene.com ↗
The study met its primary endpoint with 29% of infants (12/41; p<0.0001) sitting without support for 5 seconds by month 12, as assessed by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). |
| 2020-04-07 | Hoffmann-La Roche | Regulatory Roche provides regulatory update on risdiplam for the treatment of spinal muscular atrophy (SMA) roche.com ↗
Basel, 07 April 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY), today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. |
| 2020-04-07 | Genentech, Inc. | Regulatory Genentech Provides Regulatory Update on Risdiplam for the Treatment of Spinal Muscular Atrophy (SMA) gene.com ↗
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam with a decision expected by August 24, 2020. |
| 2020-04-07 | PTC Therapeutics | Regulatory PTC Announces Regulatory Update on Risdiplam for Spinal Muscular Atrophy (SMA) ptcbio.com ↗
PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the U.S. Food and Drug Administration (FDA) has extended the Prescription Drug User Fee Act (PDUFA) date for its review of the New Drug Application (NDA) of risdiplam to August 24, 2020. |
| 2020-02-06 | Hoffmann-La Roche | Results Roche’s risdiplam showed significant improvement in motor function in people aged 2-25 with type 2 or 3 spinal muscular atrophy roche.com ↗
The study showed that change from baseline in the primary endpoint of the Motor Function Measure scale (MFM-32)1 was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). |
| 2020-02-06 | Genentech, Inc. | Results Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 With Type 2 or 3 Spinal Muscular Atrophy gene.com ↗
The study showed that change from baseline in the primary endpoint of the Motor Function Measure 32 scale (MFM-32)* was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). |
| 2020-02-06 | PTC Therapeutics | Results SUNFISH Pivotal Results for Risdiplam (RG7916) Demonstrate Medically Meaningful Benefit in Broadest Age Group of Patients Studied with Type 2 and 3 Spinal Muscular Atrophy ptcbio.com ↗
The study demonstrated that the change from baseline in the primary endpoint Motor Function Measure scale (MFM-32) was significantly greater in patients receiving risdiplam compared to placebo (1.55 point mean difference; p=0.0156) at 12 months of treatment. |
| 2020-01-23 | Hoffmann-La Roche | Results Roche’s Risdiplam meets primary endpoint in pivotal FIREFISH trial in infants with type 1 spinal muscular atrophy roche.com ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced positive topline results from the pivotal Part 2 of the FIREFISH study, evaluating risdiplam in infants aged 1-7 months with Type 1 spinal muscular atrophy (SMA). |
| 2020-01-22 | Genentech, Inc. | Results Genentech’s Risdiplam Meets Primary Endpoint in Pivotal FIREFISH Trial in Infants With Type 1 Spinal Muscular Atrophy gene.com ↗
Risdiplam demonstrated statistically significant and medically meaningful motor milestone improvement in infants with Type 1 SMA |
| 2019-11-25 | Hoffmann-La Roche | Regulatory FDA grants priority review to Roche’s risdiplam for spinal muscular atrophy roche.com ↗
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) and granted Priority Review for risdiplam, an investigational survival motor neuron-2 (SMN-2) splicing modifier for SMA. |
| 2019-11-24 | Genentech, Inc. | Regulatory FDA Grants Priority Review to Genentech’s Risdiplam for Spinal Muscular Atrophy gene.com ↗
FDA Grants Priority Review to Genentech’s Risdiplam for Spinal Muscular Atrophy |
| 2019-11-11 | Hoffmann-La Roche | Results Roche’s risdiplam meets primary endpoint in pivotal SUNFISH trial in people with type 2 or 3 spinal muscular atrophy roche.com ↗
The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. |
| 2019-11-10 | Genentech, Inc. | Results Genentech’s Risdiplam Meets Primary Endpoint In Pivotal SUNFISH Trial in People With Type 2 or 3 Spinal Muscular Atrophy gene.com ↗
The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. |
| 2019-05-07 | Hoffmann-La Roche | Results Roche presents data from the risdiplam pivotal FIREFISH and SUNFISH studies in spinal muscular atrophy at the 2019 AAN Annual Meeting roche.com ↗
In the dose-finding Part 1 of FIREFISH, infants with Type 1 spinal muscular atrophy survive and achieve key milestones beyond those expected in the natural history of the disease |
| 2019-05-06 | Genentech, Inc. | Results Genentech Presents Data from the Risdiplam Pivotal FIREFISH and SUNFISH Studies in Spinal Muscular Atrophy at the 2019 AAN Annual Meeting gene.com ↗
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced new data from the dose-finding Part 1 of the pivotal FIREFISH trial showing infants with Type 1 spinal muscular atrophy (SMA) achieved key motor milestones after one year of treatment with investigational risdiplam. |
| 2019-04-29 | Hoffmann-La Roche | Results New Roche data at the 2019 AAN Annual Meeting showcase breadth and promise of neuroscience portfolio roche.com ↗
Risdiplam data from Part 1 of pivotal FIREFISH study show infants with Type 1 spinal muscular atrophy achieve key motor milestones and improved survival after one year of treatment |
| 2019-04-28 | Genentech, Inc. | Results New Genentech Data at the 2019 AAN Annual Meeting Showcase Breadth and Promise of Neuroscience Portfolio gene.com ↗
Risdiplam data from Part 1 of pivotal FIREFISH study show infants with Type 1 spinal muscular atrophy achieve key motor milestones and improved survival after one year of treatment |
| 2018-12-17 | Hoffmann-La Roche | Regulatory PRIME designation granted by European Medicines Agency for Roche's risdiplam for treatment of spinal muscular atrophy (SMA) roche.com ↗
Basel, 17 December 2018 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency (EMA) has granted PRIME (PRIority MEdicines) designation for the company's investigational oral medicine risdiplam (RG7916) for the treatment of people with SMA. |
| 2018-10-03 | Hoffmann-La Roche | Results Roche announces new data for risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress roche.com ↗
In the FIREFISH study in Type 1 SMA, six out of 14 infants (43%) were able to sit (with or without support), including three (21%) who achieved unassisted stable sitting after eight months of treatment. |
| 2018-10-03 | Genentech, Inc. | Results Genentech Announces New Data for Risdiplam in Spinal Muscular Atrophy (SMA) at the World Muscle Society Congress gene.com ↗
In the FIREFISH study in Type 1 SMA, six out of 14 infants (43 percent) were able to sit (with or without support), including three (21 percent) who achieved unassisted stable sitting after eight months of treatment. |
All press releases naming this drug 103 releases
| Date | Issuer | Release |
|---|---|---|
| Show all 103 releases opens the full record — a long page | ||
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 NCT03779334 ↗ |
| 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 |