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

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 Date01 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 Oct 2025·Neurology-Genetics

Single Nucleotide SMN1 Variants in a Cohort of Individuals With Spinal Muscular Atrophy

Article

Author: Gagliardi, Delia ; Albamonte, Emilio ; Meneri, Megi ; Ottoboni, Linda ; Comi, Giacomo Pietro ; Corti, Stefania ; Tiziano, Francesco Danilo ; Rimoldi, Martina ; Ronchi, Dario ; Mercuri, Eugenio ; Magri, Francesca ; Ada SANSONE, Valeria

Background and Objectives:

Spinal muscular atrophy 5q (SMA) is a motor neuron disorder caused by recessive pathogenic variants in the SMN1 gene, which encodes the survival motor neuron (SMN) protein. While the majority of patients with SMA exhibit homozygous deletions in SMN1, a minority (2%-5%) of patients with SMA harbor an SMN1 deletion plus a single nucleotide variant on the second allele, which can be identified through direct gene sequencing. The comprehensive characterization of patients with SMA is increasingly crucial considering emerging therapies and newborn screening initiatives.

Methods:

Over the past 20 years, we confirmed a molecular diagnosis of SMA in 149 patients consisting of 138 postnatal and 11 prenatal cases, through a quantitative molecular approach (real-time PCR and/or multiplex ligation-dependent probe amplification) associated with direct sequencing.

Results:

We identified homozygous SMN1 deletions in 142 probands (95%). The remaining 7 patients (5%) displayed heterozygous SMN1 deletion in compound with a different molecular defect. Notably, 1 patient presented with an intronic variant necessitating mRNA transcript analysis, a process that extended the time to diagnosis.

Discussion:

The identification of small pathogenic variants in patients with SMA is of paramount importance for enhancing diagnosis and prognosis, deciphering response variations to existing treatments, and designing novel therapies tailored to address these genetic variants. We propose a paradigm shift from current guidelines, particularly for patients with a heterozygous SMN1 deletion and a clinically compatible SMA phenotype, especially when reduced SMN transcript levels are evident. In such cases, expedited therapy initiation, including reversible treatments like nusinersen or risdiplam, is recommended without waiting for the completion of the molecular testing, thus minimizing delays in crucial therapeutic interventions.

09 Sep 2025·JCI Insight

Prenatal SMN-dependent defects in translation uncover reversible primary cilia phenotypes in spinal muscular atrophy.

Article

Author: Groen, Ewout Jn ; Genovese, Federica ; Pronot, Marie ; Viero, Gabriella ; Gillingwater, Thomas H ; Wishart, Thomas M ; Faller, Kiterie Me ; Donzel, Deborah ; Huang, Yu-Ting ; Paganin, Martina ; Signoria, Ilaria ; Cousin, Michael A ; Chaytow, Helena ; Lai, Nicole Ch ; Morris, Kimberley J ; Sharma, Gaurav ; Kline, Rachel A ; Motyl, Anna Al

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by low levels of SMN protein. Several therapeutic approaches boosting SMN are approved for human patients, delivering remarkable improvements in lifespan and symptoms. However, emerging phenotypes, including neurodevelopmental comorbidities, are being reported in some treated SMA patients, indicative of alterations in brain development. Here, using a mouse model of severe SMA, we revealed an underlying neurodevelopmental phenotype in SMA where prenatal SMN-dependent defects in translation drove disruptions in non-motile primary cilia across the central nervous system (CNS). Low levels of SMN caused widespread perturbations in translation at embryonic day (E) 14.5 targeting genes associated with primary cilia. The density of primary cilia in vivo, as well as cilial length in vitro, was significantly decreased in prenatal SMA mice. Proteomic analysis revealed downstream perturbations in primary cilia-regulated signalling pathways, including Wnt signalling. Cell proliferation was concomitantly reduced in the hippocampus of SMA mice. Prenatal transplacental therapeutic intervention with SMN-restoring risdiplam rescued primary cilia defects in SMA mouse embryos. Thus, SMN protein is required for normal cellular and molecular development of primary cilia in the CNS. Early, systemic treatment with SMN-restoring therapies can successfully target neurodevelopmental comorbidities in SMA.

01 Sep 2025·LANCET NEUROLOGY

Safety and efficacy of apitegromab in nonambulatory type 2 or type 3 spinal muscular atrophy (SAPPHIRE): a phase 3, double-blind, randomised, placebo-controlled trial

Article

Author: Masson, Riccardo ; Sansone, Valeria A ; Nance, Jessica ; Krueger, Jena ; Bruno, Claudio ; Castellano, Inmaculada Pitarch ; Seferian, Andreea M ; Mathews, Katherine ; Servais, Laurent ; Marantz, Jing L ; Rossello, Jose ; Kuntz, Nancy ; Mazurkiewicz-Beldzinska, Maria ; Kölbel, Heike ; Perlman, Seth Javier ; Finkel, Richard S ; Proud, Crystal ; Deconinck, Nicolas ; Young, Sally Dunaway ; Song, Guochen ; van der Pol, W Ludo ; Cances, Claude ; Ramos-Platt, Leigh Marie ; Blaschek, Astrid ; Fryer, Robert ; Nazario, Adelie Navas ; Holzwarth, Dorothea ; Bernes, Saunder ; Childs, Anne-Marie ; Tesi-Rocha, Carolina ; Daron, Aurore ; Baranello, Giovanni ; Tirucherai, Giridhar S ; Messina, Sonia ; Yum, Sabrina ; Crawford, Thomas O ; Butterfield, Russell J ; Kwon, Jennifer ; de la Banda, Marta Gomez Garcia ; Davion, Jean-Baptiste B. ; Statland, Jeffrey M. ; Yao, Bert ; Mercuri, Eugenio ; Richardson, Randal ; Zaidman, Craig ; Pechmann, Astrid ; Parsons, Julie ; De Waele, Liesbeth ; Batley, Kaitlin ; Mazzone, Elena Stacy ; Zhao, Guolin ; Phan, Han ; Darras, Basil T ; Nascimento Osorio, Andres ; Connolly, Anne M. ; Kotulska-Jozwiak, Katarzyna ; Steinborn, Barbara ; Cartwright, Michael ; Rady, Chamindra Laverty

BACKGROUND:

Approved spinal muscular atrophy therapies greatly improve clinical outcomes; however, substantial motor function deficits persist. Apitegromab, a fully human monoclonal antibody, selectively inhibits myostatin activation, improving muscle function. We aimed to assess the safety and efficacy of apitegromab in patients with nonambulatory type 2 or type 3 spinal muscular atrophy receiving nusinersen or risdiplam.

METHODS:

SAPPHIRE, a double-blind, placebo-controlled, phase 3 trial, was done in 48 hospitals in Belgium, France, Germany, Italy, Poland, Spain, the Netherlands, the UK, and the USA. Eligible participants were aged 2-21 years, had genetically documented SMN-deficient nonambulatory type 2 or type 3 spinal muscular atrophy, an estimated life expectancy greater than 2 years, Hammersmith Functional Motor Scale-Expanded (HFMSE) scores 10-45, and had received at least 10 months' nusinersen or at least 6 months' risdiplam therapy at screening. Participants aged 2-12 years were randomly assigned 1:1:1 to receive apitegromab 20 mg/kg, apitegromab 10 mg/kg, or placebo every 4 weeks; participants aged 13-21 years were randomly assigned 2:1 to receive apitegromab 20 mg/kg or placebo every 4 weeks. All participants, parents or caregivers, investigators, and site personnel were unaware of the treatment assignment. The primary endpoint, change from baseline in HFMSE at 12 months, was assessed in participants aged 2-12 years who received at least one dose of apitegromab or placebo and had at least one post-baseline evaluable HFMSE assessment (modified intention-to-treat set). Comparisons of the combined apitegromab dose (20 mg/kg and 10 mg/kg) versus placebo and the 20 mg/kg dose versus placebo were done with a mixed-effects model with repeated measurement. Safety was analysed in all participants who received at least one dose of apitegromab or placebo through evaluation of adverse events, physical examinations, vital signs and cardiac assessments, laboratory evaluations, and concomitant medications. SAPPHIRE is registered with ClinicalTrials.gov, NCT05156320, and is completed.

FINDINGS:

From March 28, 2022, to Sept 4, 2024, we enrolled 188 patients (156 in the population aged 2-12 years and 32 in the population aged 13-21 years); of whom 128 participants received apitegromab and 60 participants received placebo. At 12 months, least squares mean difference in HFMSE change from baseline was 1·8 (95% CI 0·30 to 3·32, p=0·019) points for participants aged 2-12 years receiving apitegromab versus placebo (least squares mean 0·6 vs -1·2). Least squares mean difference in HFMSE change from baseline was 1·4 (95% CI -0·34 to 3·13; p=0·11) for apitegromab 20 mg/kg versus placebo (least squares mean 0·2 vs -1·2). The incidence and severity of adverse events were similar between apitegromab and placebo, and consistent with spinal muscular atrophy and background spinal muscular atrophy therapy. The most frequently reported adverse events were pyrexia (apitegromab, 33 [26%] of 128 vs placebo, 17 [28%] of 60), nasopharyngitis (32 [25%] vs 14 [23%]), cough (30 [23%] vs 12 [20%]), vomiting (29 [23%] vs ten [17%]), upper respiratory tract infection (28 [22%] vs 18 [30%]), and headache (27 [21%] vs 12 [20%]). No patients discontinued due to adverse events.

INTERPRETATION:

Participants in the apitegromab treatment groups (combined 20 mg/kg and 10 mg/kg dose) achieved statistically significant improvements in motor function compared with placebo; however, the least squares mean difference was not significant between apitegromab 20 mg/kg and placebo. Overall, SAPPHIRE results build on findings from the phase 2 TOPAZ trial, showing improved motor function with a generally well tolerated safety profile, supporting the use of muscle-targeting therapy for spinal muscular atrophy.

FUNDING:

Scholar Rock.

228

News (Medical) associated with Risdiplam

23 Sep 2025

·www.fiercepharma.com

FDA rejects Scholar Rock's SMA drug, citing issues at Novo's Catalent Indiana site

The FDA has rejected Scholar Rock's application for approval of spinal muscular atrophy treatment apitegromab, citing issues with a third-party manufacturing site now owned by Novo Nordisk. Scholar Rock’s bid to become the first company to win FDA approval for a muscle-targeted treatment for spinal muscular atrophy (SMA) has been tripped up by a manufacturing issue.The FDA sent a complete response letter (CRL) to the Massachusetts biotech, rejecting its application for approval of apitegromab. The U.S. regulator cited observations stemming from a general site inspection of a third-party contract manufacturing facility in Indiana, which Novo Nordisk acquired from Catalent as part of its parent company's acquisition of the CDMO last year.The issues that were identified were not specific to the production of apitegromab, Scholar Rock said, adding that the CRL did not cite any FDA concerns relating to the efficacy or safety of the drug, which is an infused monoclonal antibody treatment. During its second quarter earnings presentation last month, Scholar Rock warned investors of the likelihood of an FDA rejection, citing issues at two third-party contract manufacturers, including one that was unnamed and performs drug substance manufacturing of apitegromab. On Tuesday, Scholar Rock said that the CRL did not identify any problems with that portion of apitegromab’s production.In early August, the facility, which Scholar Rock referred to as Catalent Indiana, submitted a response seeking to address the FDA's concerns. Following that submission, the site has continued to take corrective action and update the FDA accordingly, Scholar Rock said. When the issues are resolved, Scholar Rock will resubmit its Biologics License Application (BLA), with the expectation that the U.S. regulator will “act expeditiously,” the company said. The original target date for the FDA decision was Monday. “We are continuing to work closely with Catalent Indiana on the FDA’s manufacturing observations so that we can resubmit the apitegromab BLA as soon as possible,” David Hallal, Scholar Rock’s chairman and CEO, said in a release.Problems with Novo Nordisk’s large-scale fill-finish facility, which is in Bloomington, Indiana, are nothing new. In 2023, an FDA decision on Regeneron’s high-dose version of Eylea was delayed for two months because of issues at the plant.Last month, during its second quarter presentation, Regeneron said that two more FDA decisions relating to Eylea were being held up because of problems uncovered at the Bloomington facility in a July inspection. The company also cited the issues as the reason for an FDA rejection of its blood cancer bispecific odronextamab.In a $16.5 billion deal that was completed in December of last year, Novo Nordisk's parent Novo Holdings bought out Catalent. The transaction included Novo Holdings’ sale of three Catalent plants—one of them the Bloomington site—to Novo Nordisk for $11 billion.As for Scholar Rock, the delay affects an asset that it hopes to convert into its first marketed product. The 13-year-old company reported a cash balance of $295 million as of June 30, with its runway extending into 2027.Regulators in Europe also are reviewing an application for approval of apitegromab, with Scholar Rock expecting to launch there in 2026. Jefferies analyst Amy Li projects peak sales for the anti-myostatin drug to reach $1.8 billion.Because of its ability to preserve muscle loss, apitegromab is seen as a potential treatment to pair with obesity drugs. In June, Scholar Rock presented results from a phase 2 proof-of-concept study that showed that combining apitegromab with Eli Lilly’s tirzepatide provided a 15% loss of muscle compared to a 30% loss for patients using tirzepatide and placebo after 24 weeks. Apitegromab has been shown to improve motor function in SMA patients who are also receiving treatments that prevent motor neuron loss associated with the disease but do not directly address muscle deterioration. These medicines, all blockbusters, include Novartis’ Zolgensma, Biogen’s Spinraza and Roche’s Evrysdi.“Muscle strength and motor function are significant unmet needs for many in the SMA community and are fundamental to independence,” Kenneth Hobby, President of Cure SMA, said in the release. “A gain in motor function can allow someone to participate in important activities of daily living from self-care to work and social interactions, and as such, we urgently await the availability of the first-ever treatment with the potential to address the muscular component of SMA.”

Clinical ResultPhase 2AcquisitionDrug Approval

23 Sep 2025

·firstwordpharma.com

Biogen hit with setback in pursuit of high-dose Spinraza approval

With competition heating up in the spinal muscular atrophy (SMA) space, Biogen has been hoping FDA approval of a new dosing regimen for Spinraza (nusinersen) could help refresh interest in the antisense oligonucleotide (ASO) — but its market plans have been put on pause after the US regulator declined to greenlight the supplemental application. Biogen said Tuesday that it received a complete response letter (CRL) for a high-dose Spinraza regimen that would lower the number of loading doses to two, rather than four, followed by higher maintenance doses every four months.The letter did not flag any problems with Spinraza's data package, Biogen said, which was based on results from the Phase II/III DEVOTE study, shared at last year's World Muscle Society (WMS) meeting. The trial met its primary endpoint in treatment-naïve, symptomatic infants, who achieved significantly greater improvements in motor function than untreated matched controls.Instead, the FDA is requesting a technical information update be included in the chemistry, manufacturing and controls (CMC) portion of the drug's application. Issues related to CMC are the most frequent reason for an FDA rejection, a recent FirstWord analysis found. The agency in July posted a batch of CRLs associated with more than 200 new drug applications, and deficiencies at manufacturing facilities or subpar quality of the manufactured drug product led to a combined 60% of all rejections (see – Vital Signs: What we learned from the FDA's CRL data dump).Luckily for Biogen, the fix appears to be easy — it said the FDA has shared options to resolve the application's issue, with a resubmission planned "promptly.""While this outcome was unexpected, we remain committed to bringing the high dose regimen to people living with SMA," said Priya Singhal, head of development at Biogen. The company noted that high-dose Spinraza was recently approved in Japan, and it's currently under review in the EU. High-dose Spinraza was one of two new SMA options expected to win FDA approval this week — and Scholar Rock's entrant, apitegromab, met a similar fate to Biogen's ASO (see – Spotlight On: How will newcomers reshape the SMA market?).Separately on Tuesday, the US regulator issued a CRL to Scholar Rock's application for apitegromab, citing CMC issues. The letter identified problems with Catalent's Indiana fill-finish facility, and were not specific to the antibody. Once the issues at the facility have been addressed, Scholar Rock said it will resubmit its application for apitegromab with the hope that the FDA "will be able to act expeditiously" on the re-filing.If Biogen and Scholar Rock are successful with their resubmissions, their products will compete with Roche's oral small molecule Evrysdi (risdiplam), which has charted a steep growth trajectory since its launch in mid-2020. Last year, the more conveniently-dosed oral drug posted higher revenue figures than both the standard dosing regimen of Spinraza and Novartis' gene therapy Zolgensma (onasemnogene abeparvovec) (see – Vital Signs: Roche outmanoeuvres another gene therapy).Biogen and partner Ionis Pharmaceuticals also have another SMA shot-on-goal in the works. Registrational studies are being prepped for salanersen after Phase I data for the ASO — administered intrathecally once per year — demonstrated that it can slow neurodegeneration in patients previously treated with Zolgensma.

Gene TherapyPhase 1Drug ApprovalOligonucleotide

03 Sep 2025

·pharma.economictimes.indiatimes.com

GST waiver for 36 drugs; formulations under 5% slab

New Delhi: Executing the much-awaited overhaul of the Goods and Services Tax (GST) regime, the GST Council has brought a major relief for patients by reducing the indirect tax on medicines to 5 per cent, along with exempting as many as 36 life-saving medicines and other critical drugs. The GST council—a statutory body highest decision making body on indirect taxes led by the Union Finance Ministry at its 56th meeting in the national capital has recommended to reduce the GST on all drugs and medicines (finished formulations) from 12 per cent to 5 per cent under the new two-tier tax structure of 5 and 18 per cent slabs to be effective from September 22. Additionally the body has also recommended to exempt GST on 33 lifesaving drugs and on 3 other medicines used for treatment of cancer, rare diseases and other severe chronic diseases. Speaking at a press briefing after the council 10.5-hour long marathon meet, Finance Minister Nirmala Sitharaman said, “all recommendations have been cleared with zero reservations and have corrected various inverted duty structure problems, classification related issues and that this rationalization exercise will bring predictability and stability of GST.” “These reforms have been carried out with a focus on the common man, and after a rigorous evaluation process in most sectors including healthcare, the rates have come down drastically, she added. Previously, medicines in India were taxed at 5 per cent for life-saving and essential drugs, and at 12 per cent for others but following the announcement all medicines will attract a standard duty of 5 per cent with extensions for several medicines specified at the end of this article. Welcoming the move, Sudarshan Jain, Secretary General, IPA said, “the decision to exempt life-saving and cancer medicines from GST, is a step that will bring direct relief to patients and their families. and, the reduction in GST on a wide range of medicines from 12 per cent to 5 per cent will further help to ease the overall treatment burden and make essential therapies more affordable.” Meanwhile, for companies, the clarity on a 5 per cent slab and exemptions removes long-standing ambiguity, enabling transparent pricing and better market planning. The move will expand access in semi-urban and rural markets, ease litigation, and free up resources for innovation, said Manoj Mishra, Partner and Tax Controversy Management Leader, Grant Thornton Bharat LLP. Notably, on the issue of inverted duty involving APIs and key starting materials—which are currently taxed at 18 per cent against 12 or 5 per cent for finished formulations—further clarity is awaited from the industry. ETPharma earlier reported that, in a letter to the Finance Minister, the national chemist body, the All India Organisation of Chemists and Druggists (AIOCD) had urged, “to bring all medicines including vitamins, probiotics, nutritional, food supplements under the 5 per cent GST slab. The rate rationalization comes days after Prime Minister Narendra Modi had pitched for introducing next-gen reforms across sectors to “increase ease of living for the common man and strengthen the economy.” However, this rationalization brings a revenue shortfall challenge for governments with states likely to bear a greater share of the burden, as they receive over 40 percent of the inter-state inflows in addition to their own SGST collections. Responding to a media query at the media briefing Arvind Shrivastava, Revenue Secretary said that, based on the consumption of FY23-24 the ministry has estimated that the net revenue implication of this proposal will be around Rs 48,000 crore. “However the exercise will trigger buoyancy effects and will have a positive impact on consumer and compliance. And therefore we believe this proposal will be fiscally sustainable both for centers and the states,” Shrivastava noted. ' S.no. Drug Indication Brand name and (Company) 1 Onasemnogene abeparvovec SMA (One-time gene therapy) Zolgensma (Novartis) 2 Asciminib Blood Cancer Scemblix (Novartis) 3 Mepolizumab Asthma Nucala (GSK) 4 Pegylated Liposomal Irinotecan Pancreatic Cancer Onivyde (Ipsen) 5 Daratumumab Cancer Darzalex (J&J subsidiary) 6 Daratumumab subcutaneous Cancer Darzalex Faspro (J&J subsidiary) 7 Teclistamab Blood Cancer Tecvayli (J&J subsidiary) 8 Amivantamab Lung Cancer Rybrevant (J&J subsidiary) 9 Alectinib Lung Cancer Alecensa (Roche) 10 Risdiplam SMA Evrysdi (Roche) 11 Obinutuzumab Blood Cancer Gazyva (Roche) 12 Polatuzumab vedotin Blood Cancer Polivy (Roche) 13 Entrectinib Cancer Rozlytrek (Roche) 14 Atezolizumab Cancer (PD-L1 immunotherapy) Tecentriq (Roche) 15 Spesolimab GPP flares Spevigo (Boehringer Ingelheim) 16 Velaglucerase Alpha Gaucher Vpriv (Takeda) 17 Agalsidase Alfa Fabry disease Replagal (Takeda) 18 Rurioctocog Alpha Pegol Hemophilia Adynovate (Takeda) 19 Idursulphatase Type II Mucopolysaccharidosis Elaprase (Takeda) 20 Alglucosidase Alfa Pompe Myozyme (Sanofi) 21 Laronidase Type I Mucopolysaccharidosis Aldurazyme (Sanofi) 22 Olipudase Alfa ASMD Xenpozyme (Sanofi) 23 Tepotinib Lung Cancer Tepmetko (Maerck KGaA) 24 Avelumab Cancer (PDL-1 immunotherapy) Bavencio (Pfizer) 25 Emicizumab Hemophilia Hemlibra (Roche subsidiary) 26 Belumosudil cGVHD - 27 Miglustat Gaucher Zavesca (J&J subsidiary) 28 Velmanase Alfa Alpha-mannosidosis - 29 Alirocumab Cholesterol lowering drug (LDL-C) Praluent (Sanofi) 30 Evolocumab Cholesterol lowering drug (LDL-C) Repatha (Amgen) 31 Cystamine Bitartrate Cystagon - 32 CI-Inhibitor injection Hereditary Angioedema - 33 Inclisiran Cholesterol lowering drug (LDL-C) Leqvio (Novartis) 34 Agalsidase Beta Fabry disease Fabrazyme (Sanofi) 35 Imiglucerase Gaucher Cerezyme (Sanofi) 36 Eptacog alfa activated recombinant coagulation factor VIIa Hemophilia NovoSeven Note: Above is the list of medicines exempted from GST tax recommended by the 56th GST Council Meet/ Source: PIB By Abhijeet Singh ,

Gene Therapy

100 Deals associated with Risdiplam

External Link

KEGG	Wiki	ATC	Drug Bank
D11406	Risdiplam	M09A	DB15305

R&D Status

Approved

10 top approved records.

to view more data

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

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	United States	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	China	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Japan	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Belgium	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Brazil	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Croatia	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	France	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Italy	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Poland	Hoffmann-La Roche, Inc.	23 Dec 2016
HMN (Hereditary Motor Neuropathy) Proximal Type I	Phase 2	Russia	Hoffmann-La Roche, Inc.	23 Dec 2016

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (full treated population)	nywidbmtko(zooxbuumdv) = dljcyggcgw kbwnlzimhm (gkjuxpeexm ) View more	Positive	11 Feb 2025
NCT03779334 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy	26	EVRYSDI (2 copies of SMN2)	nywidbmtko(zooxbuumdv) = fjerzexpms kbwnlzimhm (gkjuxpeexm )	Positive	11 Feb 2025
NCT02913482 (FDA_CDER) Manual	Phase 2	HMN (Hereditary Motor Neuropathy) Proximal Type I	62	EVRYSDI	mvslzhwxxl(zngxpcrnwk) = nbkfcwidnb zsreqyclcg (ozascaoarw ) View more	Positive	11 Feb 2025
NCT02908685 (FDA_CDER) Manual	Phase 2	Spinal Muscular Atrophy \| Muscular Atrophy, Spinal, Type II	180	EVRYSDI	vncqypckrn(ycswgfxkcl) = kakvhjapnv futarmwkoq (yoceriaveu, 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	vncqypckrn(ycswgfxkcl) = pgdqiarpbn futarmwkoq (yoceriaveu, -1.22 to 0.84) View more	Positive	11 Feb 2025
NCT03779334 (RAINBOWFISH, GlobeNewswire) Manual	Phase 2	Spinal Muscular Atrophy SMN2 copies	23	Evrysdi	epxzrkbgep(yfhlpzzgid) = All of the children treated with Evrysdi who had three or more SMN2 copies (n=18), achieved standing and walking (100%). aymamsmbvk (ualdumlgmf ) Met View more	Positive	14 Oct 2024
NCT02913482 (FIREFISH, NEWS) Manual	Not Applicable	Spinal Muscular Atrophy	-	Evrysdi	jlnomesuiz(doahxckozl) = julumqymhc frwntbcoos (pnafdslxaj ) View more	Positive	14 Jun 2024
NCT03779334 (RAINBOWFISH, MDA2024) Manual	Phase 3	Spinal Muscular Atrophy SMN2 copies \| ulnar CMAP amplitude	26	Risdiplam	fsoggaxdia(iiseeunbuw) = gbnaadsijs eggnsueqsb (rlevlwspnc ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy SMN2 copies	-	Risdiplam	sbtcbrdlhn(qjgqfhsnhp) = One infant had a single overdose 10x intended dosing without related sequelae agwmebvvxz (bbkybhbxzr ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec (OA)	uvnkbydxkc(butbfoyysh) = jxpyqegvno ajkwltacpv (nclmattjvq )	-	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam (EVRYSDI)	uvnkbydxkc(butbfoyysh) = kqyzvgrkdr ajkwltacpv (nclmattjvq )	-	03 Mar 2024
NCT02908685 (SUNFISH, MDA2023) Manual	Phase 2/3	Spinal Muscular Atrophy	180	risdiplam	dotyzkjjvb(zftloxtffs) = change from baseline in the 32-item Motor Function Measure (MFM32) total score in patients treated with risdiplam (n=120) versus placebo (n=60) was met at Month 12. pzuiklxsgx (pgvjqhvqat ) Met	Positive	19 Mar 2023
NCT02908685 (SUNFISH, MDA2023) Manual	Phase 2/3	Spinal Muscular Atrophy	180	placebo		Positive	19 Mar 2023
MDA2023	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam	zxurcygduy(jwinlxsjro) = wmaddudont asxfgwdhfx (qinjwgzbkj, 22) View more	-	19 Mar 2023

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

AI Agents Built for Biopharma Breakthroughs

Accelerate discovery. Empower decisions. Transform outcomes.

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, PatSnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis

Risdiplam

Overview

Basic Info

Structure/Sequence

Related

External Link

R&D Status

Clinical Result

Translational Medicine

Deal

Core Patent

Clinical Trial

Approval

Regulation