RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), PRIME (European Union), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Accelerated assessment (European Union), Priority Review (China), Special Review Project (China)

Structure/Sequence

Molecular FormulaC22H23N7O

InChIKeyASKZRYGFUPSJPN-UHFFFAOYSA-N

CAS Registry1825352-65-5

Clinical Trials associated with Risdiplam

NCT07047144

/ RecruitingPhase 2

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

This double-blind, Phase 2, multiple-dose study will be conducted to evaluate the PK/PD, efficacy, safety, and tolerability of apitegromab in subjects <2 years old with 5q autosomal recessive SMA who have delayed motor milestones for their age attributed to SMA at the discretion of the Investigator or a Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) score <55.

Start Date15 Sep 2025

Sponsor / Collaborator

Scholar Rock, Inc.

NCT05861999

/ RecruitingPhase 4

A Phase IV Open-Label 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

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered in pediatric participants with SMA and 2 SMN2 copies who previously received onasemnogene abeparvovec and experience a plateau or decline in function. Participants to be enrolled are children <2 years of age genetically diagnosed with SMA.

Start Date14 Aug 2024

Sponsor / Collaborator-

NCT05861986

/ RecruitingPhase 4

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy

This is an open-label, single-arm, multicenter clinical study to evaluate the effectiveness and safety of risdiplam administered as an early intervention in pediatric participants with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec. Participants are children < 2 years of age genetically diagnosed with SMA.

Start Date30 May 2024

Sponsor / Collaborator-

100 Clinical Results associated with Risdiplam

100 Translational Medicine associated with Risdiplam

100 Patents (Medical) associated with Risdiplam

257

Literatures (Medical) associated with Risdiplam

01 Feb 2026·CPT-Pharmacometrics & Systems Pharmacology

Population Physiologically‐Based Pharmacokinetic Modeling to Determine Ontogeny: A Quantitative Clinical Pharmacology Example in Pediatric Rare Disease

Review

Author: Cleary, Yumi ; Gertz, Michael ; Ogungbenro, Kayode ; Prasad, Bhagwat ; Galetin, Aleksandra

ABSTRACT:

Pediatric physiologically‐based pharmacokinetic (PBPK) modelling plays an increasing role in selecting doses in children and addressing clinical pharmacology questions. Ethical concerns often limit clinical pharmacology studies that have no direct therapeutic benefit in children, highlighting the value of PBPK model predictions. However, regulatory acceptance of pediatric PBPK models remains limited because of uncertainties in system‐specific information and inadequate model qualification. Ambiguous ontogeny data of drug metabolizing enzymes (DME) and transporters are recognized as significant obstacles to the accurate pharmacokinetics (PK) prediction in children and the leading cause of insufficient pediatric PBPK model qualification. To address this challenge, a population PBPK modeling approach is proposed. This method is analogous to whole‐body PBPK modeling and allows the estimation of DME/transporter ontogenies using sparse PK data collected from children and adults by nonlinear mixed‐effect modeling. Well‐characterized ontogeny functions of key DME/transporters enhance the extrapolation ability of PBPK models and facilitate model‐informed drug development (MIDD) in children. This article proposes a strategy for pediatric PK extrapolation using population PBPK modeling, illustrated through the case example of risdiplam, approved for the treatment of spinal muscular atrophy. The ontogeny modeling, extrapolations of PK to unstudied pediatric populations, and drug–drug interaction (DDI) risk assessment are also discussed. The population PBPK modeling approach is intended to address the inconsistencies in ontogeny data and augment PBPK modeling for quantitative clinical pharmacology assessments in children. It will accelerate optimal dose finding and provide guidance for adequate use of drugs in pediatric patients, which is especially important for developing treatments for progressive pediatric rare diseases.

01 Feb 2026·BRAIN & DEVELOPMENT

Reply to “Comment on ‘Survival motor neuron protein is the optimal biomarker for evaluating the risdiplam treatment’”

Article

Author: Yokomura, Mamoru ; Otsuki, Noriko ; Saito, Kayoko ; Kato, Tamaki

01 Jan 2026·JOURNAL OF NEUROLOGY

Safety and effectiveness of risdiplam in adults with spinal muscular atrophy: a systematic review

Review

Author: Alonge, Paolo ; Gadaleta, Giulio ; Urbano, Guido

Abstract:

Background:

Risdiplam has broadened therapeutic options for spinal muscular atrophy (SMA). While its efficacy and safety are well established in children, data in adults remain limited. This review summarizes current evidence on risdiplam use in the adult SMA population.

Methods:

Following PRISMA 2020 guidelines, we systematically reviewed PubMed, Scopus, and the Cochrane Library up to September 2025 for studies including risdiplam-treated adults (≥ 18 years). Outcomes were summarized across motor, bulbar, respiratory, patient-reported, safety, and adherence domains.

Results:

Fourteen studies (> 200 adults, mainly SMA types 2 and 3) were included. Most participants were non-ambulant and treatment naïve. Motor function was generally stable, with modest yet significant improvements on RULM, HFMSE, or MFM-32, particularly among younger/less severely affected adults. Swallowing, speech, and fatigue often improved even in advanced disease. Patient-reported outcomes consistently indicated perceived gains in quality of life across all phenotypes. Adverse events were mostly mild and transient—mainly gastrointestinal symptoms, photosensitivity, or liver enzyme elevations—with very few temporary discontinuations.

Conclusions:

Risdiplam shows a favorable safety profile and provides both disease stabilization and multidimensional benefits across all functional phenotypes in adults with SMA, although further longitudinal studies using standardized outcome measures are needed to clarify its long-term impact.

251

News (Medical) associated with Risdiplam

19 Feb 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

– Full-year 2025 product and royalty revenue of $831M, exceeding guidance – – Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively – – Cash of $1.95B as of December 31, 2025 – WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025. "We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven." Key Corporate Highlights Full-year 2025 product and royalty revenue of $831 million, exceeding guidance Global launch of Sephience off to strong start Q4 2025 revenue of $92 million, including $81 million revenue in the US and $11 million revenue ex-US Sephience total net revenue of $111 million in 2025 since launch 946 total patients on commercial therapy worldwide as of December 31, 2025 1,134 patient start forms received in the US as of December 31, 2025 Approval in Japan in December 2025; approval in Brazil in February 2026 Sephience global footprint expected to increase to 20 to 30 countries by end of 2026 In December 2025, PTC sold the remainder of its Evrysdi® (risdiplam) royalty to Royalty Pharma for $240 million upfront and up to $60 million in sales-based milestones; PTC maintains the right to receive a $150 million milestone from Roche based on single-year Evrysdi sales of $2.5 billion End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington's disease (HD) program Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026 FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich's ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group Fourth Quarter and Full Year 2025 Financial Highlights Total revenues were $164.7 million for the fourth quarter of 2025, compared to $213.2 million for the fourth quarter of 2024. Total revenues were $1,730.7 million for full year 2025, compared to $806.8 million for full year 2024. Included in total revenues is collaboration and license revenue of $998.4 million for the full year 2025, related to the votoplam license and collaboration agreement with Novartis, which closed in January 2025. Total net product revenues were $184.0 million for the fourth quarter of 2025, compared to $150.1 million for the fourth quarter of 2024. Total net product revenues were $586.7 million for full year 2025, compared to $582.1 million for full year 2024. Translarna™ (ataluren) net product revenues were $39.0 million for the fourth quarter of 2025, compared to $89.1 million for the fourth quarter of 2024. Translarna net product revenues were $235.3 million for full year 2025, compared to $321.1 million for full year 2024. Emflaza® (deflazacort) net product revenues were $27.1 million for the fourth quarter of 2025, compared to $50.5 million for the fourth quarter of 2024. Emflaza net product revenues were $146.4 million for full year 2025, compared to $207.2 million for full year 2024. Roche reported Evrysdi full year 2025 sales of approximately 1,757 CHF million, resulting in royalty revenue of $244.2 million to PTC for full year 2025, compared to $203.9 million to PTC for full year 2024. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $133.1 million for the fourth quarter of 2025, compared to $124.8 million for the fourth quarter of 2024. GAAP R&D expenses were $455.2 million for full year 2025, compared to $534.5 million for full year 2024. Non-GAAP R&D expenses were $124.3 million for the fourth quarter of 2025, excluding $8.8 million in non-cash, stock-based compensation expense, compared to $116.0 million for the fourth quarter of 2024, excluding $8.8 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $419.6 million for full year 2025, excluding $35.7 million in non-cash, stock-based compensation expense, compared to $497.9 million for full year 2024, excluding $36.6 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $96.9 million for the fourth quarter of 2025, compared to $84.7 million for the fourth quarter of 2024. GAAP SG&A expenses were $347.1 million for full year 2025, compared to $300.9 million for full year 2024. Non-GAAP SG&A expenses were $87.2 million for the fourth quarter of 2025, excluding $9.7 million in non-cash, stock-based compensation expense, compared to $76.3 million for the fourth quarter of 2024, excluding $8.4 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $308.3 million for full year 2025, excluding $38.9 million in non-cash, stock-based compensation expense, compared to $262.9 million for full year 2024, excluding $38.0 million in non-cash, stock-based compensation expense. Net loss was $135.0 million for the fourth quarter of 2025, compared to net loss of $65.9 million for the fourth quarter of 2024. Net income was $682.6 million for full year 2025, compared to net loss of $363.3 million for full year 2024. Cash, cash equivalents, and marketable securities were $1,945.4 million on December 31, 2025, compared to $1,139.7 million on December 31, 2024. Shares issued and outstanding as of December 31, 2025, were 81,474,366. Full Year 2026 Financial Guidance Total product revenue of $700 to $800 million, representing a 19 to 36% increase from 2025, with the majority from Sephience GAAP R&D and SG&A expense of $775 to $815 million Non-GAAP R&D and SG&A expense of $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the call by phone, please click here to register and you will be provided with dial-in details. The webcast conference call can be accessed on the Investors section of the PTC website at . A replay of the call will be available after completion of the call and will be archived on the company's website. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Instagram and Facebook. For more Information Investors Ellen Cavaleri +1 (615) 618-6228 [email protected] Media Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "Full Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the U.S. on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Financial StatementPhase 3License out/inDrug ApprovalNDA

06 Feb 2026

·www.fiercepharma.com

Biogen pins SMA recovery on high-dose Spinraza amid projected 2026 revenue slump

Biogen hopes to gain approval of the new high-dose Spinraza formulation in the U.S. in April. Biogen recorded declining sales for Spinraza in each of the four quarters of 2025, but the Massachusetts drugmaker sees positive signs with the recent launch of its high-dose (HD) version of the spinal muscular atrophy (SMA) treatment in Japan.With HD Spinraza already approved in Europe and Biogen facing an FDA decision on the new formulation on April 3, the company has high hopes for U.S. approval and similar launch trajectories.“We’re certainly off to a better start than we expected. It’s not just in terms of adoption, it’s also switch backs,” CEO Chris Viehbacher said in a quarterly conference call Friday. “We certainly have seen much higher levels of efficacy in the study, which suggests that there’s an increased benefit from getting to a therapeutic level faster.”After its FDA approval in 2016, Spinraza’s sales scaled up quickly, topping out at $2.1 billion in 2019. However, they’ve dropped to $1.55 billion last year, the company reported Friday. The decline throughout 2025 was especially precipitous, from $424 million in the first quarter to $356 million in the fourth, which fell well below analysts’ consensus projection from Visible Alpha of $385 million.Competition from Roche’s SMA oral treatment Evrysdi, which was initially approved in 2020, has taken a toll. Last week the Swiss company reported Evrysdi sales at 1.76 billion Swiss francs ($2 billion). A year ago to the month, Roche scored an FDA nod for a tablet version of the treatment, gaining another convenience edge on infused Spinraza and Novartis’ gene therapy blockbuster Zolgensma, which also has seen a sales dip. In discussions with physicians, Viehbacher said that the key question they face in treating SMA patients is “efficacy versus convenience.”“If you ask them about efficacy, most of them believe it’s really Spinraza, but at some point, the convenience of the oral starts to attract patients,” Viehbacher said. “Here, we’re going to be dramatically increasing the level of efficacy, and I think the choice between oral and efficacy will be harder for some physicians and parents.”As for the company’s performance overall in 2025, revenue reached $9.9 billion for a 2% year-over-year uptick. It was the first time since 2019 that Biogen saw an annual sales increase. The company generated revenue that year of $14.4 billion.Biogen does not expect the growth to continue in 2026, however, projecting a mid-single-digit decline to a range of between $9.3 billion and $9.5 billion. The trend was reflected in fourth-quarter overall sales, which came in at $2.28 billion for a 7% year-over-year decline and a sequential slide from $2.54 billion. When asked when to expect Biogen to return to growth, Viehbacher said much would depend on positive phase 3 results from two products which would be potentially launched in 2028. Until then, headwinds to growth include erosion in Europe for multiple sclerosis (MS) treatment Tecfidera and the recent introduction of biosimilar competition for another Biogen MS drugs, Tysabri.Viehbacher also is considering M&A.“We continue to look for potential acquisition opportunities. These are companies with post-phase 3 results or are early stage in their commercialization, certainly no more than around the $5-to-$6-billion mark,” Viehbacher said. “There are certainly companies out there, but we haven’t found one that we can acquire for a price that we think makes sense for our shareholders, but we continue to look. We are looking every day in the marketplace.”

AcquisitionDrug ApprovalPhase 3

30 Dec 2025

·firstwordpharma.com

Royalty Pharma locks in full royalty stream from Roche's Evrysdi

Royalty Pharma has agreed to pay $240 million upfront to acquire the final portion of PTC Therapeutics' remaining royalty interest in Roche's spinal muscular atrophy drug Evrysdi (risdiplam). The transaction also includes up to $60 million in potential sales-based milestones.Specifically, the deal will see Royalty Pharma control 100% of a tiered royalty on worldwide net sales of the drug, entitling it to 8% on sales up to $500 million, 11% on sales between $500 million and $1 billion, 14% on sales between $1 billion and $2 billion, and 16% on sales exceeding $2 billion.Roche reported revenues of CHF 1.6 billion ($2 billion) for Evrysdi in 2024, up 18% at constant exchange rates, and analysts project the orally administered SMN2 splicing modifier could reach CHF 2.3 billion ($2.9 billion) in annual revenue by 2030.Royalty Pharma said it will begin receiving the increased royalty in the first quarter of 2026 based on fourth-quarter 2025 sales.Cleared by the FDA in 2020, Evrysdi is now approved in more than 100 countries and used by over 21,000 patients worldwide. This week's agreement builds on Royalty Pharma's expanding exposure to the drug. In 2020, it acquired roughly 43% of PTC's royalty interest for $650 million, and in late 2023 it paid $1 billion upfront for an additional stake while securing options to purchase more.In 2024, PTC recorded $203.9 million in royalty revenue from Evrysdi, up from $168.9 million the prior year.

Drug ApprovalLicense out/in

100 Deals associated with Risdiplam

External Link

KEGG	Wiki	ATC	Drug Bank
D11406	Risdiplam	M09A	DB15305

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Spinal Muscular Atrophy	United States	Genentech, Inc.	07 Aug 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Genetic Diseases, Inborn	Phase 3	Italy	F. Hoffmann-La Roche Ltd.	03 Jan 2022
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 3	Italy	F. Hoffmann-La Roche Ltd.	03 Jan 2022
Juvenile Spinal Muscular Atrophy	Phase 3	Italy	F. Hoffmann-La Roche Ltd.	03 Jan 2022
Muscular Atrophy, Spinal, Type II	Phase 3	Italy	F. Hoffmann-La Roche Ltd.	03 Jan 2022
Spinal Muscular Atrophies of Childhood	Phase 2	United States	Roche China Holding Ltd.	27 Sep 2016
Spinal Muscular Atrophies of Childhood	Phase 2	United States	F. Hoffmann-La Roche Ltd.	27 Sep 2016
Spinal Muscular Atrophies of Childhood	Phase 2	Japan	F. Hoffmann-La Roche Ltd.	27 Sep 2016
Spinal Muscular Atrophies of Childhood	Phase 2	Japan	Roche China Holding Ltd.	27 Sep 2016
Spinal Muscular Atrophies of Childhood	Phase 2	Belgium	Roche China Holding Ltd.	27 Sep 2016
Spinal Muscular Atrophies of Childhood	Phase 2	Belgium	F. Hoffmann-La Roche Ltd.	27 Sep 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03032172 (Jewelfish, CTgov)	Phase 2	Spinal Muscular Atrophy	174	Risdiplam	nniypzzxnm(fuqmyyxsaq) = tngfwzazov egtnxsylmp (ipcktjpcvf, xrdvoxqxto - rlqqvafgac) View more	-	01 Oct 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (full treated population)	johmqzaaqv(evlxwueofi) = gvnknrhxea vqpibcxoyc (pwlilmpbgo ) View more	Positive	11 Feb 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (2 copies of SMN2)	johmqzaaqv(evlxwueofi) = wbuvpytadx vqpibcxoyc (pwlilmpbgo )	Positive	11 Feb 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	EVRYSDI	lyhglpkbpl(mmvxqmaaor) = jtufwxitbk dizltfowrx (fbkhooucko, 0.61 - 2.11) View more	Positive	11 Feb 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	Placebo	lyhglpkbpl(mmvxqmaaor) = ytezlbhkxl dizltfowrx (fbkhooucko, -1.22 to 0.84) View more	Positive	11 Feb 2025
NCT02913482 (FDA_CDER) Manual	Phase 2	HMN (Hereditary Motor Neuropathy) Proximal Type I	62	EVRYSDI	cgytmhjhib(eulnosxamu) = bwzwqpugna ggyvkndumu (qhpwkvmgdr ) View more	Positive	11 Feb 2025
NCT03779334 (RAINBOWFISH, GlobeNewswire) Manual	Phase 2	Spinal Muscular Atrophy SMN2 copies	23	Evrysdi	jhtizeeitb(sepzrlsnqy) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). jjnmbshenr (atfowwtyyg ) Met View more	Positive	14 Oct 2024
NCT02913482 (FIREFISH, NEWS) Manual	Not Applicable	Spinal Muscular Atrophy	-	Evrysdi	jshpktqpiq(fzwctmpdex) = pyzimwykgk ohihtlgvqt (hjsttaprox ) View more	Positive	14 Jun 2024
NCT03779334 (RAINBOWFISH, CTgov)	Phase 2	Spinal Muscular Atrophy	26	risdiplam (2 SMN2 Copies, Risdiplam)	wtuqkuifqu = ifjnjscjrc jaqptmgghq (dbhkcomyjv, dpqncdoqwj - hvilbwnpdh) View more	-	05 Mar 2024
NCT03779334 (RAINBOWFISH, CTgov)	Phase 2	Spinal Muscular Atrophy	26	risdiplam (3 SMN2 Copies, Risdiplam)	mlyvmreqgy(ggdvcilswx) = wulktimrih xbvyiviman (dtbegcopjm, xviubxpdcv - kfvhjkrniz) View more	-	05 Mar 2024
NCT03779334 (RAINBOWFISH, MDA2024) Manual	Phase 3	Spinal Muscular Atrophy SMN2 copies \| ulnar CMAP amplitude	26	Risdiplam	fxikededid(ybgmldqiwa) = qktrpuylpv bftfjfskqa (ckpftobjnb ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy SMN2 copies	-	Risdiplam	tsoqnxyfim(ucyfphjljf) = One infant had a single overdose 10x intended dosing without related sequelae juckaeozrt (xzmkswuaun ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec (OA)	zrorbyoyen(kjgpbblith) = klsvdjbtfe rudiazgygb (nmkvkfnmpi )	-	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam (EVRYSDI)	zrorbyoyen(kjgpbblith) = spwjnefarw rudiazgygb (nmkvkfnmpi )	-	03 Mar 2024

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