RegulationOrphan Drug (US), Orphan Drug (KR), Orphan Drug (AU), Priority Review (AU), Accelerated assessment (EU), Priority Review (CN), Special Review Project (CN), PRIME (EU), Fast Track (US), Orphan Drug (EU)

Structure

Molecular FormulaC22H23N7O

InChIKeyASKZRYGFUPSJPN-UHFFFAOYSA-N

CAS Registry1825352-65-5

Clinical Trials associated with Risdiplam

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

Hoffmann-La Roche, Inc.

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

Hoffmann-La Roche, Inc.

NCT05808764 / RecruitingPhase 2

A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy

This study will evaluate the pharmacokinetics (PK) and safety of risdiplam in participants with spinal muscular atrophy (SMA) under 20 days of age at first dose.

Start Date26 Apr 2024

Sponsor / Collaborator

Hoffmann-La Roche, Inc.

100 Clinical Results associated with Risdiplam

100 Translational Medicine associated with Risdiplam

100 Patents (Medical) associated with Risdiplam

165

Literatures (Medical) associated with Risdiplam

01 Dec 2024·Lancet Regional Health-Europe

Efficacy and safety of gene therapy with onasemnogene abeparvovec in children with spinal muscular atrophy in the D-A-CH-region: a population-based observational study

Article

Background:

Real-world data on gene addition therapy (GAT) with onasemnogene abeparvovec (OA), including all age groups and with or without symptoms of the disease before treatment are needed to provide families with evidence-based advice and realistic therapeutic goals. Aim of this study is therefore a population-based analysis of all patients with SMA treated with OA across Germany, Austria and Switzerland (D-A-CH).

Methods:

This observational study included individuals with Spinal Muscular Atrophy (SMA) treated with OA in 29 specialized neuromuscular centers in the D-A-CH-region. A standardized data set including WHO gross motor milestones, SMA validated motor assessments, need for nutritional and respiratory support, and adverse events was collected using the SMArtCARE registry and the Swiss-Reg-NMD. Outcome data were analyzed using a prespecified statistical analysis plan including potential predictors such as age at GAT, SMN2 copy number, past treatment, and symptom status.

Findings:

343 individuals with SMA (46% male, 54% female) with a mean age at OA of 14.0 months (range 0-90, IQR 20.0 months) were included in the analysis. 79 (23%) patients were clinically presymptomatic at the time of treatment. 172 (50%) patients received SMN2 splice-modifying drugs prior to GAT (risdiplam: n = 16, nusinersen: n = 154, both: n = 2). Functional motor improvement correlated with lower age at GAT, with the best motor outcome in those younger than 6 weeks, carrying 3 SMN2 copies, and being clinically presymptomatic at time of treatment. The likelihood of requiring ventilation or nutritional support showed a significantly increase with older age at the time of GAT and remained stable thereafter. Pre-treatment had no effect on disease trajectories. Liver-related adverse events occurred significantly less frequently up to 8 months of age. All other adverse events showed an even distribution across all age and weight groups.

Interpretation:

Overall, motor, respiratory, and nutritional outcome were dependent on timing of GAT and initial symptom status. It was best in presymptomatic children treated within the first six weeks of life, but functional motor scores also increased significantly after treatment in all age groups up to 24 months. Additionally, OA was best tolerated when administered at a young age. Our study therefore highlights the need for SMA newborn screening and immediate treatment to achieve the best possible benefit-risk ratio.

Funding:

The SMArtCARE and Swiss-Reg-NMD registries are funded by different sources (see acknowledgements).

01 Nov 2024·MUSCLE & NERVE

Safety of risdiplam in spinal muscular atrophy patients after short‐term treatment with nusinersen

Article

Author: Feng, Yijie ; Jiang, Liya ; Jin, Jianing ; Mao, Shanshan ; Yan, Yue

Abstract:

Introduction/Aims:

Following the approval of risdiplam, there are more possibilities for disease‐modifying therapy (DMT) in children with spinal muscular atrophy (SMA). Non‐treatment‐naïve subjects with SMA involved in the JEWELFISH study, designed to evaluate the safety and tolerability of risdiplam, were required to undergo a washout period before receiving risdiplam. This study aims to investigate the safety of administering risdiplam in patients within 90 days of receiving treatment with nusinersen.

Methods:

Data were collected on SMA patients who had undergone treatment with nusinersen, and who then received risdiplam within 90 days of their last dose of nusinersen, including demographic characteristics, information on treatment with nusinersen and risdiplam, adverse events, and laboratory assessments in a follow‐up period of 90 days, presented as median (range).

Results:

A total of 15 children with SMA were reported, including 8 males and 7 females. The median number of doses of previous nusinersen treatment received was 8 (6–17) doses, and the median age at first risdiplam treatment was 4.3 (1.9–11.2) years. Specifically, 8 children received risdiplam 30 days or less after their most recent nusinersen treatment, 2 at 31–60 days after nusinersen, and 5 at 61–89 days post‐nusinersen. Adverse events of pyrexia, pneumonia, vomiting and rash were reported in 4 patients.

Discussion:

Our study showed good safety data on patients who received risdiplam following nusinersen within the washout period of 90 days. This supplements the JEWELFISH study in the era of DMT, providing additional guidance for clinicians, but additional data from other centers is needed.

01 Nov 2024·MUSCLE & NERVE

Nutrition outcomes of disease modifying therapies in spinal muscular atrophy: A systematic review

Review

Author: O'Brien, Katie ; Woodcock, Ian R. ; Nguo, Kay ; Yiu, Eppie M. ; Billich, Natassja ; Davidson, Zoe E.

Abstract:

The nutritional implications of spinal muscular atrophy (SMA) are profound. Disease modifying therapies (DMT) have improved clinical outcomes. This review describes the impact of DMT on nutrition outcomes. A systematic search strategy was applied across seven databases until May 2023. Eligible studies measured nutrition outcomes in individuals with SMA on DMT (nusinersen, risdiplam or onasemnogene abeparvovec [OA]) compared to untreated comparators. Nutrition outcomes included anthropometry, feeding route, swallowing dysfunction, dietary intake, dietetic intervention, nutritional biochemistry, metabolism, gastrointestinal issues and energy expenditure. Articles retrieved were screened in duplicate, data were extracted and appraised systematically. Sixty three articles from 54 studies were included; 41% (n = 22) investigated nusinersen in pediatric participants with SMA type 1. Anthropometry (n = 18), feeding route (n = 39), and swallowing dysfunction (n = 18) were the most commonly reported outcomes. In combined pediatric and adult cohorts, BMI z‐score remained stable post nusinersen therapy. The proportion of children with SMA requiring enteral nutrition was stable post nusinersen therapy. Ability to thrive at age 1.5 years was higher in children treated in early infancy with OA compared to historical controls. Significant heterogeneity existed across study population characteristics and outcome measures. Nusinersen may prevent deterioration in some nutrition outcomes; and OA in early infancy may be associated with improved nutrition outcomes. Timing of DMT initiation is an important consideration for future nutrition research. Studies investigating nutrition as a primary outcome of DMT, using consistent outcome measures are required for nutritional management strategies for this cohort to be appropriately tailored.

129

News (Medical) associated with Risdiplam

23 Oct 2024

·www.globenewswire.com

[Ad hoc announcement pursuant to Art. 53 LR] Roche’s strong sales growth of 9% (CER) continues in the third quarter of 2024; Group sales increase 6% in the first nine months

Group sales grew by 6%1 at constant exchange rates (CER) (2% in CHF) in the first nine months, driven by the high demand for both our medicines and diagnostics; excluding COVID-19-related products, sales increased by 8%In the third quarter, Group sales rose by 9% (6% in CHF), as they did in the second quarter Pharmaceuticals Division sales rose by 7% in the first nine months; the strong growth of 9% in the base business2 was driven by continued high demand for our newer medicines to treat severe diseases; Vabysmo (serious eye diseases), Phesgo (breast cancer) and Ocrevus (multiple sclerosis) were major growth driversDiagnostics Division sales increased by 5% in the first nine months, while the base business2 grew by 8% due to higher demand for immunodiagnostic, pathology and molecular solutionsHighlights: US approval for Itovebi (inavolisib) for breast cancer, Ocrevus Zunovo subcutaneous injection for multiple sclerosis and Tecentriq Hybreza subcutaneous formulation for various types of cancerEU approval for Vabysmo for retinal vein occlusion (RVO), a serious eye disease, and PiaSky for paroxysmal nocturnal haemoglobinuria (PNH), a rare life-threatening blood conditionPositive phase III data for Gazyva/Gazyvaro (lupus nephritis, a kidney disease), Xofluza (influenza) and Tecentriq (lung cancer). New positive phase II data for fenebrutinib (multiple sclerosis), and new positive long-term data for Evrysdi (spinal muscular atrophy)Acquired AntlerA Therapeutics for a novel target in ophthalmology, and signed agreement for the acquisition of two next-generation CDK inhibitor drugs targeting breast cancer from Regor PharmaceuticalsClosing of acquisition of LumiraDx’s point-of-care technology to expand access to diagnostic testing in primary care and low- and middle-income countriesLaunch of the cobas Respiratory flex test, the first to use our new Temperature-Activated Generation of Signal (TAGS) technologyWHO endorsement for CINtec PLUS testing for cervical cancer prevention Outlook for 2024 confirmed Roche CEO Thomas Schinecker: “Our strong growth momentum continued in the third quarter, reflecting the high demand for our innovative medicines and diagnostic solutions and their positive impact on patients’ lives around the world. We made significant progress in our pharmaceuticals portfolio in the last quarter with five important regulatory approvals for our medicines, three positive phase III read-outs, and two acquisitions to strengthen our oncology and ophthalmology pipelines. Itovebi (inavolisib) recently received US approval based on clinical data demonstrating a reduction of more than 50% in the risk of death or worsening disease for people suffering from a form of advanced, hard-to-treat breast cancer. In addition, we had positive phase III results for Gazyva/Gazyvaro in lupus nephritis, a potentially life-threatening kidney disease for which limited treatment options are available today. We confirm our outlook for 2024.” Sales CHF millions As % of sales % change January‒September 2024 2023 2024 2023 At CER In CHF Group 44,984 44,053 100.0 100.0 6 2 Pharmaceuticals Division 34,257 33,372 76.2 75.8 7 3 United States 18,166 17,430 40.4 39.6 7 4 Europe 6,613 6,259 14.7 14.2 7 6 Japan 2,083 2,937 4.6 6.7 -21 -29 International* 7,395 6,746 16.5 15.3 19 10 Diagnostics Division 10,727 10,681 23.8 24.2 5 0 All figures shown in the table were restated to reflect the shift of the Foundation Medicine (FMI) business from the Pharmaceuticals Division to the Diagnostics Division. *Asia-Pacific, CEETRIS (Central Eastern Europe, Türkiye, Russia and Indian subcontinent), Latin America, Middle East, Africa, Canada, others Outlook for 2024 confirmed Roche expects an increase in Group sales in the mid single digit range (CER). Core earnings per share are targeted to grow in the high single digit range (CER), excluding the impact from the resolution of tax disputes in 2023. Roche expects to further increase its dividend in Swiss francs. Group salesIn the first nine months of 2024, Group sales increased by 6% at CER (2% in CHF) to CHF 45.0 billion as strong demand for our novel medicines as well as diagnostic products including immunodiagnostic, pathology and molecular solutions more than offset the anticipated decline in COVID-19-related sales and the impact of biosimilar/generic erosion. The appreciation of the Swiss franc against most currencies had an adverse impact on the sales reported in Swiss francs compared to constant exchange rates. The Pharmaceuticals Division sales increased by 7% to CHF 34.3 billion, while the base business (excluding COVID-19) grew by 9%, driven primarily by higher sales of Vabysmo (severe eye diseases), Phesgo (breast cancer), Ocrevus (multiple sclerosis), Hemlibra (haemophilia) and Polivy (blood cancer). These five medicines together generated total sales of CHF 13.2 billion, an increase of CHF 2.7 billion (CER) from the first nine months of 2023. The eye medicine Vabysmo, launched in early 2022, continues to be a major growth driver, generating sales of CHF 2.8 billion on growing demand in all regions. Sales of Avastin (various types of cancer), Herceptin (breast and gastric cancer) and MabThera/Rituxan (blood cancer, rheumatoid arthritis) decreased by a combined CHF 0.5 billion as the impact of biosimilar competition slowed further. Sales of the COVID-19 medicine Ronapreve were negligible compared with CHF 0.5 billion in the first nine months of 2023. In the United States, sales grew by 7% as strong sales of Vabysmo, Ocrevus, Polivy and Xolair (food allergies) were partially offset by the continued decline in sales of medicines for which patent protection has expired. Vabysmo achieved CHF 2.1 billion in sales, showing a high uptake in both new patients and patients switching from other medications. In Europe, sales rose by 7%, driven by demand for Vabysmo as well as by the continued uptake of Phesgo, Ocrevus, Evrysdi (spinal muscular atrophy) and Hemlibra. This was partially offset by lower sales of medicines for which patent protection has expired and of Perjeta (breast cancer) due to the ongoing conversion of patients to Phesgo. Sales in Japan were down 21%, mainly due to the base effect of the supply of Ronapreve (COVID-19) to the government in the first quarter of 2023. Excluding this effect, sales in Japan were 3% lower as strong demand for Phesgo and Vabysmo was more than offset by the impact of government price cuts and lower sales of medicines for which patent protection has expired. Sales in the International region surged by 19%, led by demand for Perjeta, Hemlibra, Tecentriq (cancer immunotherapy), Phesgo and Ocrevus as well as the launch of Elevydis (gene therapy, Duchenne muscular dystrophy). Sales in China increased by 8%, driven by Xofluza, Perjeta, Polivy and Avastin. The Diagnostics Division sales increased by 5% to CHF 10.7 billion, while the base business (excluding COVID-19) grew by 8%. Immunodiagnostic products, which include cardiac, oncology and thyroid tests, were the main growth drivers (10%). Additional growth came from pathology and molecular solutions. Sales of COVID-19 tests were CHF 0.1 billion in the first nine months of 2024 compared with CHF 0.4 billion in the corresponding period last year. Sales growth was reported across regions, with the Europe, Middle East and Africa (EMEA) region growing by 5%, North America by 6%, Asia-Pacific by 2% and Latin America by 18%. Pharmaceuticals: key developments Compound Milestone Regulatory Itovebi (inavolisib)Breast cancer FDA approves Itovebi, a targeted treatment for advanced hormone receptor (HR)-positive, HER2-negative breast cancer with a PIK3CA mutation Approval is based on phase III INAVO120 results, showing the regimen based on Itovebi (inavolisib) more than doubled progression-free survival compared with palbociclib and fulvestrant alone in the first-line settingThis approval helps address an urgent unmet need in breast cancer for people with a mutated PIK3CA gene, one of the most commonly mutated genes in HR-positive disease and which is associated with poor prognosisItovebi is Roche’s first targeted therapy approved for people with HR-positive disease, the most prevalent breast cancer subtype, marking an important step in our ambition to continue bringing innovative medicines to more people with breast cancer More information: Media Release, 11 October 2024 Ocrevus ZunovoMultiple sclerosis FDA approves Ocrevus Zunovo as the first and only twice-a-year 10-minute subcutaneous injection for people with relapsing and progressive multiple sclerosis Ocrevus Zunovo has the potential to expand treatment options to centres without intravenous (IV) infrastructure or with IV constraints, like at a doctor’s officeThis approval is backed by a decade of proven safety and efficacy data of Ocrevus IV, with over 350,000 people treated globallyOcrevus Zunovo offers people with multiple sclerosis more options to access treatment based on their individual needs More information: Media Release, 16 September 2024 Tecentriq Hybreza Various types of cancer FDA approves Tecentriq Hybreza, the first and only subcutaneous anti-PD-(L)1 cancer immunotherapy Tecentriq Hybreza provides patients and physicians with greater flexibility of treatment options while showing safety and efficacy consistent with intravenous (IV) TecentriqNew subcutaneous option reduces treatment time to approximately seven minutes, compared with 30‒60 minutes for IV infusion More information: Media Release, 13 September 2024 PiaSkyRare blood disease PiaSky approved in the EU as the first monthly subcutaneous treatment for people with paroxysmal nocturnal haemoglobinuria (PNH) With the option of self-administration, PiaSky (crovalimab) has the potential to reduce treatment burden for people with PNH and their caregivers in EuropeApproval is based on COMMODORE 2, where subcutaneous (SC) PiaSky once a month was equivalent to intravenous eculizumab every two weeksPiaSky advances C5 inhibition through innovative recycling technology, which enables its monthly SC administration More information: Media Release, 27 August 2024 Vabysmo Severe eye diseases European Commission approves Vabysmo for treatment of retinal vein occlusion (RVO) Approval is based on data from two phase III studies in branch and central retinal vein occlusion (RVO) showing early and sustained vision improvements non-inferior to aflibercept, and robust retinal drying with VabysmoAdditional submitted data shows that up to 60% of people receiving Vabysmo were able to extend treatment intervals to three or four monthsVabysmo is already approved in several countries, including the US and Japan, for RVO and in nearly 100 countries for people with neovascular or ‘wet’ age-related macular degeneration (nAMD) and diabetic macular edema (DME) More information: Media Release, 30 July 2024 Phase III, pivotal and other key readouts EvrysdiSpinal muscular atrophy Majority of children with spinal muscular atrophy (SMA) treated with Evrysdi are able to sit, stand and walk independently, two-year data demonstrate 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 SMAAll children were able to swallow and feed orally, with none requiring permanent ventilationEvrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, with more than 16,000 people with SMA treated globally More information: Media Release, 14 October 2024 Gazyva/GazyvaroKidney disease Positive phase III results for Gazyva/Gazyvaro show superiority to standard therapy alone in people with lupus nephritis The REGENCY study met its primary endpoint, demonstrating statistically significant and clinically meaningful treatment benefits in people with active lupus nephritisGazyva/Gazyvaro is designed to target an underlying cause of lupus nephritis, aiming to prevent or delay progression to end-stage kidney diseaseLupus nephritis is a potentially life-threatening manifestation of an autoimmune disease affecting 1.7 million people worldwide, primarily women; up to one-third of people on current treatments will progress to end-stage kidney disease within 10 years More information: Media Release, 26 September 2024 Xofluza Influenza Positive phase III results show Xofluza significantly reduces the transmission of influenza viruses Data from the CENTERSTONE study shows single-dose Xofluza reduces transmission of influenza from an infected person to household membersThis is the first time that any antiviral used in the treatment of a respiratory viral illness has demonstrated a transmission reduction benefit in a global phase III studyReducing the spread of infection in the household could help limit transmission within communities and societies, easing the burden of both seasonal and pandemic influenza on healthcare systems More information: Media Release, 19 September 2024 Fenebrutinib Multiple sclerosis Fenebrutinib demonstrated near-complete suppression of disease activity and disability progression for up to 48 weeks in patients with relapsing multiple sclerosis New phase II data show vast majority of patients experiencing no relapses or disability progressionFenebrutinib suppressed acute and chronic MRI lesionsFenebrutinib’s safety profile was consistent with previous and ongoing clinical trials across multiple diseases including more than 2,700 people to date More information: Media Release, 4 September 2024 Susvimo Severe eye disease New data for Susvimo demonstrates sustained efficacy in two serious diabetic eye conditions Two-year phase III data presented at ASRS 2024 show Susvimo’s potential as an alternative to eye injections to treat diabetic macular edema (DME) and diabetic retinopathy (DR)Safety data were consistent with the known safety profile for Susvimo in people with DME and DRAdditionally, the US FDA has accepted the filing application for Susvimo in DME and DR based on one-year Pagoda and Pavilion study data More information: Media Release, 18 July 2024 Other Pharma Research and Early Development Center Roche opens Pharma Research and Early Development Center in Basel to accelerate scientific innovation Switzerland’s most innovative research and development centre underscores Roche’s long-term investment in scientific advancement to meet patient needsThe new centre will simplify and increase collaboration, thereby accelerating scientific innovation More information: Media Release, 10 September 2024 Pharmaceuticals sales Sales CHF millions As % of sales % change January–September 2024 2023 2024 2023 At CER In CHF Pharmaceuticals Division 34,257 33,372 100.0 100.0 7 3 United States 18,166 17,430 53.0 52.2 7 4 Europe 6,613 6,259 19.3 18.8 7 6 Japan 2,083 2,937 6.1 8.8 -21 -29 International* 7,395 6,746 21.6 20.2 19 10 All figures shown in the table were restated to reflect the shift of the Foundation Medicine (FMI) business from the Pharmaceuticals Division to the Diagnostics Division. * Asia-Pacific, CEETRIS (Central Eastern Europe, Türkiye, Russia and Indian subcontinent), Latin America, Middle East, Africa, Canada, others Top-selling medicines Total United States Europe Japan International CHF m % CHF m % CHF m % CHF m % CHF m % Ocrevus Multiple sclerosis 5,056 9 3,640 7 961 11 - - 455 26 Hemlibra Haemophilia A 3,280 10 1,906 5 690 10 259 4 425 42 Vabysmo Eye diseases (nAMD, DME, RVO) 2,816 79 2,146 67 454 148 86 37 130 256 Perjeta3 Breast cancer 2,809 -1 1,029 -4 502 -17 92 -36 1,186 17 Tecentriq Cancer immunotherapy 2,703 1 1,325 -8 649 5 277 -1 452 32 Actemra/RoActemra3 RA, COVID-19 1,948 5 949 11 508 -11 225 9 266 17 Xolair3 Asthma 1,737 11 1,737 11 - - - - - - Kadcyla3 Breast cancer 1,494 6 574 4 428 -2 71 4 421 22 Evrysdi Spinal muscular atrophy 1,246 21 429 15 435 18 66 10 316 35 Phesgo Breast cancer 1,244 58 404 29 543 44 88 - 209 104 Alecensa Lung cancer 1,151 7 372 12 217 1 144 3 418 8 Herceptin3 Breast and gastric cancer 1,063 -11 201 -20 227 -15 11 -48 624 -5 MabThera/Rituxan3 Blood cancer, RA 1,023 -16 615 -17 109 -21 12 -26 287 -10 Avastin 3 Various cancer types 943 -17 289 -20 63 -17 149 -33 442 -8 Activase/TNKase3 Cardiac diseases 895 2 850 1 - - - - 45 5 Polivy Blood cancer 817 41 410 83 142 6 143 -4 122 79 Gazyva/Gazyvaro3 Blood cancer 670 13 333 15 185 8 21 -16 131 21 Pulmozyme3 Cystic fibrosis 329 0 213 -5 55 -3 1 17 60 23 Mircera3 Anaemia related to kidney disease 304 -1 - - 31 -6 28 -23 245 4 CellCept3 Immunosuppressant 283 1 17 -21 81 -13 27 -9 158 17 DME: diabetic macular edema / nAMD: neovascular or ‘wet’ age-related macular degeneration / RVO: retinal vein occlusion / RA: rheumatoid arthritis Diagnostics: key developments Product Milestone VENTANA CLDN18 assay Gastric or gastro-oesophageal junction cancer Roche obtains CE certification for the first companion diagnostic to identify patients with gastric and gastro-oesophageal junction cancer eligible for targeted treatment with VYLOY The new VENTANA CLDN18 (43-14A) RxDx assay helps fulfil an unmet medical need by enabling clinicians to identify patients with gastric or gastro-oesophageal junction (GOJ) cancer who may benefit from a targeted treatment optionCLDN18.2 is an emerging biomarker in gastric and GOJ cancers and helps predict the likelihood of response to targeted therapyAs the leader in companion diagnostics, Roche continues to build on its commitment to improve personalised healthcare to enable better patient outcomes More information: Media Release, 10 October 2024 cobas Respiratory flex testRespiratory illnesses Roche launches the first test to use its breakthrough TAGS technology for high-throughput, simultaneous detection of 12 respiratory viruses The new Temperature-Activated Generation of Signal (TAGS) technology enables up to 15 targets to be detected simultaneously in a single patient sample on the high-throughput molecular diagnostic analysers cobas 5800, 6800 and 8800TAGS has the potential to revolutionise testing for other infectious diseases in the future, by bringing high-throughput customised syndromic panel testing to the routine clinical laboratoryThe first TAGS-based test to be made available, the cobas Respiratory flex, offers fast, efficient detection of up to 12 of the most common respiratory viruses with the flexibility for targeted testing, expediting accurate diagnosis, optimising antimicrobial use and saving time in the lab More information: Media Release, 24 September 2024 CINtec PLUS Cervical cancer WHO endorses dual-stain cytology (CINtec PLUS) testing in its cervical cancer prevention guidelines, advancing patient care and underlining Roche’s role in pioneering cervical cancer solutions CINtec PLUS Cytology is the only FDA-approved and CE-marked dual-stain test to triage human papillomavirus (HPV)-positive cervical cancer screening test resultsDual-stain biomarkers aid in detection of cervical precancer and may reduce the number of women who undergo unnecessary colposcopy procedures while allowing earlier intervention for those who are at higher risk of developing cervical cancerThis recognition follows the American Society for Colposcopy and Cervical Pathology (ASCCP)’s recent inclusion of dual-stain testing in cervical cancer screening guidelines, as well as other WHO prequalifications of Roche’s cobas HPV test More information: Media Release, 23 September 2024 cobas MPXV test, LightMix Mpox Roche responds to WHO’s declaration of a global health emergency due to the ongoing mpox outbreak Roche is committed to supporting all those working to overcome the mpox outbreak by providing access to high-quality polymerase chain reaction (PCR) testingRoche confirms that its cobas MPXV test and the LightMix research-use-only kits detect the latest mpox virus variantsWe are actively working to enhance laboratory testing capacity for mpox worldwide More information: Media Release, 20 August 2024 LumiraDx Roche closes acquisition of LumiraDx’s point-of-care technology to expand access to diagnostic testing in primary care The acquisition of LumiraDx’s point-of-care technology received all required antitrust and regulatory clearancesLumiraDx’s transformative point-of-care solution will complement Roche’s diagnostics portfolio across clinical chemistry, immunochemistry, coagulation and molecular, and across multiple disease areasBy integrating LumiraDx, Roche will further its ambition to deliver decentralised solutions that expand global access to testing in primary care settings worldwide More information: Media Release, 29 July 2024 Diagnostics sales Sales CHF millions As % of sales % change January–September 2024 2023 2024 2023 At CER In CHF Diagnostics Division 10,727 10,681 100.0 100.0 5 0 Customer Areas4 Core Lab 6,052 5,836 56.4 54.6 9 4 Molecular Lab5 1,903 1,897 17.7 17.8 4 0 Near Patient Care6 1,616 1,902 15.1 17.8 -10 -15 Pathology Lab 1,156 1,046 10.8 9.8 15 11 Regions Europe, Middle East and Africa 3,589 3,569 33.5 33.4 5 1 North America5 3,222 3,103 30.0 29.1 6 4 Asia-Pacific 3,146 3,263 29.3 30.5 2 -4 Latin America 770 746 7.2 7.0 18 3 More information on Roche sales in the first nine months of 2024: Q3 2024 presentationAppendix with tables About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in diagnostics and pharmaceuticals with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com. All trademarks used or mentioned in this release are protected by law. References [1] Unless otherwise stated, all growth rates and comparisons to the previous year in this document are at constant exchange rates (CER: average rates 2023) and all total figures quoted are reported in CHF. [2] Pharmaceuticals Division base business: excluding COVID-19 medicine Ronapreve. Diagnostics Division base business: excluding COVID-19-related products. [3] Products launched before 2015. [4] Core Lab: diagnostics solutions in the areas of immunoassays, clinical chemistry and CustomBiotech. Molecular Lab: diagnostics solutions for pathogen detection and monitoring, donor screening, sexual health and genomics, genomic tumour profiling. Near Patient Care: diagnostics solutions in emergency rooms, medical practices and directly with patients, including integrated personalised diabetes management. Pathology Lab: diagnostics solutions for tissue biopsies and companion diagnostics. [5] Sales in the Molecular Lab customer area include sales from the Foundation Medicine business, which moved under the responsibility of the Diagnostics Division from the Pharmaceuticals Division effective 1 January 2024. The comparative information for 2023 has been restated accordingly. [6] Sales in the new Near Patient Care customer area include sales from Diabetes Care and the Point of Care business, both previously shown as separate customer areas. The comparative information for 2023 has been restated accordingly. Cautionary statement regarding forward-looking statements This document contains certain forward-looking statements. These forward-looking statements may be identified by words such as ‘believes’, ‘expects’, ‘anticipates’, ‘projects’, ‘intends’, ‘should’, ‘seeks’, ‘estimates’, ‘future’ or similar expressions or by discussion of, among other things, strategy, goals, plans or intentions. Various factors may cause actual results to differ materially in the future from those reflected in forward-looking statements contained in this document, such as: (1) pricing and product initiatives of competitors; (2) legislative and regulatory developments and economic conditions; (3) delay or inability in obtaining regulatory approvals or bringing products to market; (4) fluctuations in currency exchange rates and general financial market conditions; (5) uncertainties in the discovery, development or marketing of new products or new uses of existing products, including without limitation negative results of clinical trials or research projects, unexpected side effects of pipeline or marketed products; (6) increased government pricing pressures; (7) interruptions in production; (8) loss of or inability to obtain adequate protection for intellectual property rights; (9) litigation; (10) loss of key executives or other employees; and (11) adverse publicity and news coverage. The statement regarding earnings per share growth is not a profit forecast and should not be interpreted to mean that Roche’s earnings or earnings per share for this or any subsequent period will necessarily match or exceed the historical published earnings or earnings per share of Roche. Roche Global Media Relations Phone: +41 61 688 8888 / e-mail: media.relations@roche.com Hans Trees, PhD Phone: +41 79 407 72 58 Sileia Urech Phone: +41 79 935 81 48 Nathalie Altermatt Phone: +41 79 771 05 25 Lorena Corfas Phone: +41 79 568 24 95 Simon Goldsborough Phone: +44 797 32 72 915 Karsten Kleine Phone: +41 79 461 86 83 Nina Mählitz Phone: +41 79 327 54 74 Kirti Pandey Phone: +49 172 6367262 Yvette Petillon Phone: +41 79 961 92 50 Dr Rebekka Schnell Phone: +41 79 205 27 03 Roche Investor Relations Dr Bruno Eschli Phone: +41 61 68-75284 e-mail: bruno.eschli@roche.com Dr Sabine Borngräber Phone: +41 61 68-88027 e-mail: sabine.borngraeber@roche.com Dr Birgit Masjost Phone: +41 61 68-84814 e-mail: birgit.masjost@roche.com Investor Relations North America Loren Kalm Phone: +1 650 225 3217 e-mail: kalm.loren@gene.com Attachments 23102024_Roche Q32023 appendix tables 23102024_Roche Media Release Q32024_EN

Phase 3Clinical ResultDrug ApprovalAcquisitionPhase 2

14 Oct 2024

·pmlive.com

Roche presents two-year data for spinal muscular atrophy treatment Evrysdi in children

Roche has presented positive two-year results from an ongoing study of its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) in children who were treated pre-symptomatically as infants before six weeks of age. Affecting approximately one in every 10,000 babies, SMA is a severe and progressive neuromuscular disease characterised by insufficient levels of the SMN protein. Depending on the form of SMA, patients’ physical strength and their ability to walk, eat or breathe can be considerably diminished or lost. Roche’s Evrysdi, which can be administered at home in liquid form by mouth or by feeding tube, is designed to treat the disease by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues. Results from the RAINBOWFISH study presented at this year’s World Muscle Society Congress showed that 100% of Evrysdi-treated patients who had three or more SMN2 copies achieved standing and walking milestones, with most achieving these within World Health Organization windows of typical child development. Among those with two SMN2 copies, 100% could sit and 60% could stand and walk independently after two years of treatment. Additionally, all patients were able to swallow and feed orally after two years of treatment, with none requiring permanent ventilation, and children in the study showed cognitive skills typical of those without SMA. Roche’s chief medical officer and head of global product development, Levi Garraway, said the two-year findings “confirm the potential of early intervention with Evrysdi to meaningfully improve the lives of children with SMA”. “Working in tandem with newborn screening programmes, Evrysdi is the only non-invasive SMA treatment that can be administered during a child’s first hours of life,” he said. Roche leads the clinical development of Evrysdi, which is already approved in more than 100 countries, as part of a collaboration with the SMA Foundation and PTC Therapeutics. The latest results for the therapy come four months after the company presented five-year data for Evrysdi at the Cure SMA Research and Clinical Care Meeting. After five years of treatment with Evrysdi, 91% of children in the open-label extension of the FIREFISH study were alive, 81% were alive without permanent ventilation and 59% were able to sit without support for at least 30 seconds.

Clinical ResultClinical Study

13 Oct 2024

·www.gene.com

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

Sunday, Oct 13, 2024 Positive data confirm Evrysdi efficacy and safety in children first treated pre-symptomatically before 6 weeks of age, with most achieving motor milestones similar to children without SMAAll children were able to swallow and feed orally, with none requiring permanent ventilationEvrysdi is the only non-invasive SMA therapy and is approved in over 100 countries, with more than 16,000 people with SMA treated globally South San Francisco, CA -- October 13, 2024 -- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), presented positive 2-year data from the ongoing RAINBOWFISH study at the 29th World Muscle Society (WMS) Congress, October 8-12, 2024, assessing the efficacy and safety of Evrysdi® (risdiplam) in children with SMA who were treated pre-symptomatically as infants before 6 weeks of age (n=23). 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. “In children with SMA, motor neuron degeneration starts before the onset of symptoms, so time is of the essence if we hope to preserve muscle function,” said Laurent Servais, M.D., Ph.D., Professor of Paediatric Neuromuscular Diseases at the MDUK Oxford Neuromuscular Centre. “It’s heartening to see that through early intervention with Evrysdi these children have achieved important milestones like sitting, standing and walking that would typically be unattainable without treatment.” All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%) milestones as assessed by Bayley Scales of Infant and Toddler Development, Third Edition (BSID-III) and Hammersmith Infant Neurological Examination, Module 2 (HINE-2), with most achieving these milestones within World Health Organization (WHO) windows of typical child development. Among the children with two SMN2 copies (n=5), all could sit (100%) and most could stand and walk (60%) independently after 2 years of treatment. After 2 years of treatment, all children were able to swallow and feed orally and none required permanent ventilation. Natural history studies indicate that without disease-modifying treatment, children with Type 1 SMA would not be able to reach such milestones, nor typically live past the age of 2. After 2 years of Evrysdi treatment, children in the study showed cognitive skills typical of children without SMA, as assessed by the BSID-III Cognitive Scale. This study was the first clinical trial in SMA to assess cognition as an exploratory endpoint using a standardized scale. “These 2-year findings confirm the potential of early intervention with Evrysdi to meaningfully improve the lives of children with SMA,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “Working in tandem with newborn screening programs, Evrysdi is the only non-invasive SMA treatment that can be administered during a child’s first hours of life.” To assess outcomes of early treatment initiation before the onset of symptoms, children in the study started treatment with Evrysdi before 6 weeks of age (median age of first dose was 25 days). The study analyzed outcomes against the number of copies of the SMN2 gene each child had. Generally, fewer SMN2 copy numbers are associated with more severe SMA. There were no deaths or adverse events (AEs) leading to withdrawal or treatment discontinuation. The most common AEs were teething, gastroenteritis, diarrhea, eczema and pyrexia. The AEs observed in the year-2 analysis are generally consistent with those AEs seen in other Evrysdi trials in SMA. AEs were more reflective of age than underlying SMA. The majority of AEs were not considered treatment-related and resolved over time. Genentech leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. About Evrysdi® (risdiplam) Evrysdi is a survival motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor neuron (SMN) protein deficiency. Evrysdi is administered daily at home in liquid form either by feeding tube or by mouth. Evrysdi is designed to treat SMA by increasing and sustaining the production of SMN protein in the central nervous system (CNS) and peripheral tissues, as demonstrated in animal models. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and core motor functions, such as swallowing, speaking and breathing. Evrysdi was granted PRIME designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by the U.S. Food and Drug Administration in 2017. In 2021, Evrysdi was awarded Drug Discovery of the Year by the British Pharmacological Society as well as the Society for Medicines Research Award for Drug Discovery. Evrysdi is currently approved in more than 100 countries, and the dossier is under review in a further 12 countries. A new risdiplam room-temperature stable tablet is currently under review by regulators. Evrysdi is currently being, or has been, evaluated in numerous global multicenter trials in people with SMA: FIREFISH (NCT02913482) – an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Infants were approximately 5.5 months of age (median) at the time of enrollment and of the 58 infants that completed the first year of treatment, 52 entered the open-label extension study. The study met its primary endpoint and has concluded after 5 years of follow up. SUNFISH (NCT02908685) – a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. The study met its primary endpoint. JEWELFISH (NCT03032172) – an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi. The study has completed recruitment (n=174). RAINBOWFISH (NCT03779334) – an open-label, single-arm, multicenter study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of Evrysdi in babies (n=26), from birth to 6 weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms. The study met its primary endpoint. MANATEE (NCT05115110) – a Phase II/III clinical study to evaluate the safety and efficacy of GYM329 (RG6237), an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age. The FDA Office of Orphan Products Development granted GYM329 Orphan Drug Designation for the treatment of patients with SMA in December 2021. The study is currently recruiting. HINALEA 1 (NCT05861986) and HINALEA 2 (NCT05861999) – Phase IV clinical studies to evaluate the effectiveness and safety of Evrysdi in patients under 2 years of age at enrollment, who received onasemnogene abeparvovec gene therapy either pre-symptomatically or post-symptomatically, following a genetically confirmed diagnosis of 5q–autosomal recessive SMA. The studies are currently recruiting. About SMA SMA is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual’s physical strength and their ability to walk, eat or breathe can be significantly diminished or lost. What is Evrysdi? Evrysdi is a prescription medicine used to treat spinal muscular atrophy (SMA) in children and adults. Important Safety Information Before taking Evrysdi, tell your healthcare provider about all of your medical conditions, including if you: are pregnant or plan to become pregnant, as Evrysdi may harm your unborn baby. Ask your healthcare provider for advice before taking this medicine are a woman who can become pregnant: Before you start your treatment with Evrysdi, your healthcare provider may test you for pregnancy Talk to your healthcare provider about birth control methods that may be right for you. Use birth control while on treatment and for at least 1 month after stopping Evrysdi Pregnancy Registry. There is a pregnancy registry for women who take Evrysdi during pregnancy. The purpose of this registry is to collect information about the health of the pregnant woman and her baby. If you are pregnant or become pregnant while receiving Evrysdi, tell your healthcare provider right away. Talk to your healthcare provider about registering with the Evrysdi Pregnancy Registry. Your healthcare provider can enroll you in this registry or you can enroll by calling 1-833-760-1098 or visiting http://www.evrysdipregnancyregistry.com are an adult male. Evrysdi may affect a man’s ability to have children (fertility). Ask a healthcare provider for advice before taking this medicine are breastfeeding or plan to breastfeed. It is not known if Evrysdi passes into breast milk and may harm your baby Tell your healthcare provider about all the medicines you take You should receive Evrysdi from the pharmacy as a liquid. If the medicine in the bottle is a powder, do not use it. Contact your pharmacist for a replacement Avoid getting Evrysdi on your skin or in your eyes. If Evrysdi gets on your skin, wash the area with soap and water. If Evrysdi gets in your eyes, rinse your eyes with water The most common side effects of Evrysdi include: For later-onset SMA: fever diarrhea rash For infantile-onset SMA: fever diarrhea rash runny nose, sneezing, and sore throat (upper respiratory infection) lung infection (lower respiratory infection) constipation vomiting cough These are not all of the possible side effects of Evrysdi. For more information on the risk and benefits profile of Evrysdi, ask your healthcare provider or pharmacist. You may report side effects to the FDA at 1-800-FDA-1088 or http://www.fda.gov/medwatch. You may also report side effects to Genentech at 1-888-835-2555. Please see full Prescribing Information for additional Important Safety Information. For more information, go to https://www.evrysdi.com/. About Genentech in Neuroscience Neuroscience is a major focus of research and development at Genentech. Our goal is to pursue groundbreaking science to develop new treatments that help improve the lives of people with chronic and potentially devastating diseases. Genentech and Roche are investigating more than a dozen medicines for neurological disorders, including multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and Duchenne muscular dystrophy. Together with our partners, we are committed to pushing the boundaries of scientific understanding to solve some of the most difficult challenges in neuroscience today. About Genentech Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com. All trademarks used or mentioned in this release are protected by law. # # #

Clinical ResultOrphan DrugDrug ApprovalClinical Study

100 Deals associated with Risdiplam

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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	US	Genentech, Inc.	07 Aug 2020

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	US	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	US	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	JP	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	JP	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	BE	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	BE	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	BR	F. Hoffmann-La Roche Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	BR	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	HR	Roche China Holding Ltd.	19 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	HR	F. Hoffmann-La Roche Ltd.	19 Dec 2016

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03779334 (RAINBOWFISH, GlobeNewswire) Manual	Phase 2	Spinal Muscular Atrophy SMN2 copies	23	Evrysdi	npdxijwozl(uinxtvofwv) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). xqnyyqpnqz (rtsqsrmmzs ) Met View more	Positive	14 Oct 2024
FIREFISH (NEWS) Manual	Not Applicable	Spinal Muscular Atrophy	-	Evrysdi	djtbltoihm(damdugevdf) = kwtghlsfes yorrvichsg (pbjgkunlla ) View more	Positive	14 Jun 2024
NCT03779334 (RAINBOWFISH, MDA2024) Manual	Phase 3	Spinal Muscular Atrophy SMN2 copies \| ulnar CMAP amplitude	26	Risdiplam	fxwzljsymt(bkpxxxgjwx) = veuupibjrl vrhykonvyd (kcavbqouhw ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy SMN2 copies	-	Risdiplam	liqyyahazx(whjenyiozj) = One infant had a single overdose 10x intended dosing without related sequelae gleyjiozas (nbfxssngmg ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec (OA)	ydooorszwd(opvvaotdwl) = yqihjiixgw bmrnahyfxe (gyiomdwgvi )	-	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam (EVRYSDI)	ydooorszwd(opvvaotdwl) = vcuzxmlsqi bmrnahyfxe (gyiomdwgvi )	-	03 Mar 2024
MDA2023	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam-treated patients	upgbnvmfcj(enddfiidjh) = jskxxbpuxj czxxlfodwx (zyuiawodcr ) View more	-	19 Mar 2023
MDA2023	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam	auildtoivm(ceonqvjyry) = mpbttjqysl cwgjzklang (uzgzvyghot, 22) View more	-	19 Mar 2023
NCT04256265 (expanded access program, Pubmed \| Ann Clin Transl Neurol)	Not Applicable	Muscular Atrophy, Spinal, Type II	155	Risdiplam	pczjzlcbxd(dlaqkertkk) = The most frequently reported adverse events were diarrhea (n = 10; 6.5%), pyrexia (n = 7; 4.5%), and upper respiratory tract infection (n = 5; 3.2%). The most frequently reported serious adverse event was pneumonia (n = 3; 1.9%). No deaths were reported. utgiljczba (fncpecaqba )	Positive	14 May 2022
NCT02908685 (SUNFISH, Pubmed \| Lancet Neurol) Manual	Phase 3	Juvenile Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	risdiplam	hdlwbomlvo(rgarwzhvmq) = tvuheztgyj jlfeiowuod (vemzloiioy, 0.61 - 2.11) View more	Positive	01 Jan 2022
NCT02908685 (SUNFISH, Pubmed \| Lancet Neurol) Manual	Phase 3		180	Placebo	hdlwbomlvo(rgarwzhvmq) = jryuqhgfqi jlfeiowuod (vemzloiioy, -1.22 to 0.84) View more	Positive	01 Jan 2022
NCT02913482 (FIREFISH, Pubmed \| Br Dent J) Manual	Phase 2/3	Spinal Muscular Atrophy	41	Risdiplam (daily risdiplam as)	fixssxxzxz(cbifdkgplq) = dyhliurnqu rqrfqjuimw (uvkdcehcep ) View more	Positive	29 Jul 2021
NCT02913482 (FIREFISH, Pubmed \| Br Dent J) Manual	Phase 2/3	Spinal Muscular Atrophy	41	Risdiplam (no treatment in historical)	ckafsshscq(dkrphzgkou) = qkuotwqkmm kzkceiwfwg (dhpgciqymk ) View more	Positive	29 Jul 2021

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