This study aims to collect safety and efficacy data during long-term follow-up in SMA patients treated with OAV101 IV or IT. The safety and efficacy of OAV101 will be evaluated up to 15 years after administration.

Start Date30 May 2024

Sponsor / Collaborator

China Novartis Institutes for BioMedical Research Co., Ltd.

[+2]

NCT06019637

/ RecruitingNot Applicable

A Long-term Safety Study in Brazilian Patients With a Confirmed Diagnosis of Spinal Muscular Atrophy (SMA) Treated With Onasemnogene Abeparvovec (Zolgensma®) - ARISER Study

A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)

Start Date22 Nov 2023

Sponsor / Collaborator

Novartis Pharmaceuticals Corp.

IRCT20221024056284N1

/ CompletedNot ApplicableIIT

Evaluation the color match of two single shade resin composites; Charisma Diamond ONE and Charisma Topaz ONE in class five restorations and assessment of dentist and patient satisfaction

Start Date21 Jan 2023

Sponsor / Collaborator

Mashhad University of Medical Sciences

100 Clinical Results associated with Onasemnogene abeparvovec

100 Translational Medicine associated with Onasemnogene abeparvovec

100 Patents (Medical) associated with Onasemnogene abeparvovec

367

Literatures (Medical) associated with Onasemnogene abeparvovec

01 Feb 2025·EUROPEAN SPINE JOURNAL

Scoliosis development in 5q-spinal muscular atrophy under disease modifying therapies

Article

Author: Perka, Carsten ; Weiß, Claudia ; Schönnagel, Lukas ; Vu-Han, Tu-Lan ; Pumberger, Matthias ; Köhli, Paul

Abstract:

Purpose:

5q-spinal muscular atrophy (SMA) is a treatable neuromuscular disorder associated with scoliosis in up to 90% of patients. New SMA therapies could mark a paradigm shift in scoliosis management, but their effects on scoliosis development remain unclear. This study aims to observe scoliosis progression in the current treatment landscape to inform management strategies.

Methods:

We conducted a cross-sectional retrospective analysis of 94 SMA patients treated at our center. Scoliosis development was evaluated in 75 patients using spine radiographs and electronic health records. Statistical analysis was performed using Python and GraphPad Prism. One-way ANOVA and Pearson correlation were used for group comparisons and correlation analysis, respectively.

Results:

Scoliosis parameters in 5q-SMA patients who had received either nusinersen, onasemnogene abeparvovec, risdiplam, or their combinations showed mean ages at scoliosis detection were 23.94, 55.52, and 168.11 months for SMA types 1, 2, and 3, respectively. Cobb angles at detection showed no significant intergroup differences. The mean ages at scoliosis surgery were 60, 88.43, and 124.8 months. Pelvic obliquity (PO) was highest in type 1 and lowest in type 3. A strong correlation (r = 0.9) was found between PO measurement techniques. HFMSE scores correlated moderately with scoliosis severity (r = -0.38), while CHOP-INTEND showed no correlation.

Conclusion:

The observations made in this study suggest that the effects of SMA therapies do not prevent scoliosis development. The improved prognosis may lead to a growing cohort of SMA type 1 and 2 patients with early onset scoliosis who require early growth-friendly surgical interventions.

28 Jan 2025·NEUROLOGY

Safety and Efficacy of IV Onasemnogene Abeparvovec for Pediatric Patients With Spinal Muscular Atrophy

Article

Author: Moreno, Teresa ; De Waele, Liesbeth ; Muntoni, Francesco ; Baranello, Giovanni ; Quijano-Roy, Susana ; McMillan, Hugh J. ; Farrar, Michelle A. ; Bernardo Escudero, Roberto ; Mercuri, Eugenio ; Alecu, Iulian ; Straub, Volker ; Jong, Yuh-Jyh ; Freischläger, Frank ; Laugel, Vincent ; Darras, Basil T. ; Seibert, Julia ; Zaidman, Craig M. ; Chien, Yin-Hsiu

BACKGROUND AND OBJECTIVES:

Safety and efficacy of IV onasemnogene abeparvovec has been demonstrated for patients with spinal muscular atrophy (SMA) weighing <8.5 kg. SMART was the first clinical trial to evaluate onasemnogene abeparvovec for participants weighing 8.5-21 kg.

METHODS:

SMART was an open-label, multicenter, phase 3b study conducted across 13 sites in 9 countries (NCT04851873). Symptomatic pediatric participants with SMA (any type; treatment-naïve or had discontinued prior treatment) were stratified into 3 weight cohorts (≥8.5-13, >13-17, and >17-21 kg), administered onasemnogene abeparvovec, and followed for 52 weeks. Corticosteroids were initiated 24 hours before infusion with dose increases in response to adverse events (AEs) and subsequent tapering at investigator discretion. The primary objective was safety. Secondary objective was efficacy (motor function/motor milestones).

RESULTS:

Twenty-four participants were enrolled; the majority had SMA type 2 (n = 11), 3 SMN2 copies (n = 18), and prior treatment (n = 21). All participants completed the study; no deaths occurred. All participants had ≥1 treatment-related AE(s), 7 of 24 (29%) had serious treatment-related AEs, and 23 of 24 (96%) had ≥1 AE of special interest. Twenty of 24 participants (83%) had asymptomatic hepatotoxicity events, which were primarily transaminase elevations. No participant had bilirubin elevations >2× upper limit of normal, developed symptomatic hepatotoxicity, or met Hy law criteria. Transient asymptomatic thrombocytopenia events were reported in 17 of 24 participants (71%); all resolved spontaneously with no related bleeding events reported. Three of 24 participants (13%) had cardiac AEs (all unrelated to treatment). No thrombotic microangiopathy or dorsal root ganglionopathy-related AEs were reported. AE frequency and severity were similar across weight groups, although corticosteroid exposure was greater for the 2 heavier cohorts (median 135.0, 201.0, and 194.0 days, respectively) with 37% and 33% still on corticosteroids at the study end. By week 52, most participants maintained or improved motor function (Hammersmith Functional Motor Scale-Expanded 16/18; Revised Upper Limb Module 15/17); 4 participants (all 3 SMN2 copies) achieved new motor milestones.

DISCUSSION:

Onasemnogene abeparvovec safety profile was similar across weight groups in this heterogenous participant population. Frequency and duration of asymptomatic aminotransferase elevations and thrombocytopenia are notable findings. Most participants demonstrated maintenance or improvement of motor function, suggesting clinical benefit for patients with SMA weighing up to 21 kg.

TRIAL REGISTRATION INFORMATION:

ClinicalTrials.gov identifier (NCT04851873, clinicaltrials.gov/study/NCT04851873) submitted April 19, 2021. First participant enrolled on September 8, 2021.

CLASSIFICATION OF EVIDENCE:

This study provides Class IV evidence that intravenous onasemnogene abeparvovec is safe in pediatric patients with SMA who weigh 8.5-21 kg.

01 Jan 2025·PHARMACOECONOMICS

Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability

Article

Author: Vreman, Rick A ; Goettsch, Wim G ; Callenbach, Marcelien H E ; Mantel-Teeuwisse, Aukje K ; Leopold, Christine

OBJECTIVE:

To construct a framework and calculation tool to compare the consequences of implementing different payment models for high-cost, one-off potentially curative therapies and enable decision making to ultimately enhance timely patient access to innovative health interventions.

METHODS:

A framework outlining steps to determine potentially suitable payment models was developed. Based on the framework, a supporting calculation tool operationalised as an Excel-based model was constructed to quantify the associated costs for an average patient during the timeframe of the intended payment agreement, the total budget impact and associated benefits expressed in quality-adjusted life-years for the total expected lifetime of the patient population. To demonstrate the potential of the framework, three case studies were used: onasemnogene abeparvovec (Zolgensma^®), brexucabtagene autoleucel (Tecartus^®) and etranacogene dezaparvovec (Hemgenix^®). A hypothetical case study was used to illustrate the output of the calculation tool.

RESULTS:

Part 1 of the framework presents steps for matching a suitable reimbursement and payment model with the disease and treatment characteristics. The reimbursement and payment models are further specified in Part 2. Part 3 guides end users through the setup of a calculation tool with which the financial impact can be calculated of two payment models: a price discount model and an outcome-based spread payment model with a discount. Part 4 concerns the output of the calculation tool, showing how different payment models lead to different financial consequences under three assumptions of longer term effectiveness.

CONCLUSIONS:

The presented framework provides decision makers with insight into the financial consequences of their chosen payment model under different assumptions. This can aid reimbursement negotiations by clarifying the optimal choice given a therapy's characteristics.

245

News (Medical) associated with Onasemnogene abeparvovec

23 Jan 2025

·www.pharmaceutical-technology.com

Biogen’s higher dose Spinraza under review by US and EU regulators

Spinal muscular atrophy (SMA) is a rare genetic condition that can cause muscle weakness. Credit: KATERYNA KON/SCIENCE PHOTO LIBRARY via Getty Images. The US Food and Drug Administration (FDA) and European Medicines Agency (EMA) have accepted Biogen ’s applications for a higher dose regimen of spinal muscular atrophy (SMA) treatment Spinraza (nusinersen). The proposed higher dose regimen includes two 50mg doses administered 14 days apart, followed by a maintenance dose of 28mg every four months. This is fewer doses compared to the existing regimen, which involves four loading doses of 12mg over 60 days and a maintenance dose of 12mg every four months. Biogen previously announced that it was planning regulatory approval for the investigational dose regimen in September 2024, alongside data from the Phase II/III DEVOTE study which has supported these regulatory filings. The three-part study (NCT04089566) enrolled 145 patients of various ages across various SMA types. In a key cohort of 75 treatment-naïve children with infantile-onset SMA, the higher-dose regimen of Spinraza demonstrated statistically significant improvements in motor function at six months, as measured by the CHOP-INTEND scale. The results were compared to a prespecified untreated control group from the earlier ENDEAR trial , which supported Spinraza’s standard dose approval. Secondary outcomes from the DEVOTE trial showed favourable trends for the higher-dose version in biomarker and efficacy measures compared to the 12mg dose. Safety data indicated that adverse events were consistent with SMA and Spinraza’s established profile. Notably, the incidence of serious adverse events was lower in the higher dose group at 60%, compared to the 72% incidence seen in the 12mg group. Spinraza – approved in the US in 2016 and Europe in 2017 – was the first disease-modifying therapy for SMA. It is an antisense oligonucleotide (ASO) designed to increase the production of survival motor neurone (SMN) protein, addressing the underlying cause of motor neuron loss. The treatment is administered directly into the central nervous system to target motor neurons. Despite its early success, Spinraza faces increasing competition from newer SMA therapies. Roche’s Evrysdi (risdiplam), approved in the US in 2020 and the EU in 2021 , is an oral treatment that may be a more convenient way of administration. Novartis’s Zolgensma (onasemnogene abeparvovec), approved in the US in 2019 and the EU in 2020 , provides a one-time treatment option for SMA. These therapies have shifted market dynamics, impacting Spinraza’s sales. Spinraza pulled in $1.74bn in sales for Biogen in 2023, as per the company’s financials. Sales of the drug are set to decrease steadily year-on-year, generating $1.15bn in 2030, according to GlobalData’s Pharma Intelligence Center. By contrast, Evrysdi and Zolgensma are expected to generate $2.78bn and $2.13bn in 2030, respectively. The global SMA market , valued at $2.7bn in 2023, is projected to grow to $3bn by 2033 in the seven major markets (US, UK, France, Germany, Italy, Spain, and Japan). GlobalData is the parent company of Pharmaceutical Technology.

Clinical ResultDrug ApprovalOligonucleotideBiosimilar

14 Jan 2025

·www.prnewswire.com

REGENXBIO and Nippon Shinyaku Announce Exclusive Partnership to Develop and Commercialize RGX-121 and RGX-111 for MPS Diseases

REGENXBIO to receive $110 million upfront, potential milestone payments of up to $700 million and meaningful double-digit royalties on net sales, and lead manufacturing Nippon Shinyaku to lead commercialization of first potential gene therapies for Mucopolysaccharidosis II (MPS II) and Mucopolysaccharidosis I (MPS I) in U.S. and Asia REGENXBIO retains rights to RGX-121 Priority Review Voucher (PRV) with potential accelerated approval expected in 2025; rolling BLA submission underway ROCKVILLE, Md., Jan. 14, 2025 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) and Nippon Shinyaku Co., Ltd. (Nippon Shinyaku) today announced a strategic partnership for the development and commercialization of RGX-121 for the treatment of Mucopolysaccharidosis II (MPS II), also known as Hunter syndrome, and RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome. Under the terms of the agreement, REGENXBIO will receive $110 million at closing and up to an additional $700 million if certain milestones are achieved, consisting of $40 million in potential development and regulatory milestones and $660 million in potential sales milestones. REGENXBIO will also receive meaningful double-digit royalties on net sales in the U.S. and Asia (collectively, the "Licensed Territory"). "This partnership with Nippon Shinyaku is exciting in that it maximizes our collective strengths and enables access of two potentially transformational medicines to key markets," said Curran M. Simpson, President and Chief Executive Officer, REGENXBIO. "The structure of the agreement allows us to leverage our expertise in gene therapy manufacturing while also capturing milestones and a meaningful share of future product revenues. RGX-121 is poised to be the first gene therapy for MPS II with potential FDA approval as early as late 2025, and RGX-111 has demonstrated very promising results in Phase 1/2 study. With Nippon Shinyaku's expertise in rare disease and strong commercial capabilities, we look forward to working together to get both of these promising candidates across the finish line for patients." "RGX-121 and RGX-111 represent one-time gene therapies that can potentially change the course of MPS disease, and we are very pleased to be partnering with REGENXBIO, experts in gene therapy development and manufacturing," said Toru Nakai, President and Representative Director of Nippon Shinyaku. "We are confident these therapies can bring tremendous value to those living with MPS II and I." Per the agreement, Nippon Shinyaku will commercialize both products in the Licensed Territory and future clinical development of RGX-121 and RGX-111 will be led by REGENXBIO. REGENXBIO retains all rights to, and 100 percent of any proceeds related to the sale of, the Priority Review Voucher (PRV) for RGX-121 received upon potential approval. REGENXBIO will lead the manufacturing of both products for clinical and commercial supply in the Licensed Territory. REGENXBIO reserves the right to develop and commercialize these products in countries outside of the Licensed Territory. The transaction is expected to close by the end of the first quarter of 2025, subject to the customary conditions, including applicable regulatory approvals. About RGX-121 RGX-121 is a potential one-time AAV therapeutic for the treatment of boys with MPS II. RGX-121 expressed protein is structurally identical to normal I2S. Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 has received Orphan Drug Product, Rare Pediatric Disease, Fast Track and Regenerative Medicine Advanced Therapy designations from the U.S. Food and Drug Administration and advanced therapy medicinal products (ATMP) classification from the European Medicines Agency. About RGX-111 RGX-111 is designed to use the AAV9 vector to deliver the α-l-iduronidase (IDUA) gene to the central nervous system (CNS). By providing rapid IDUA delivery to the brain, RGX-111 could potentially help prevent the progression of cognitive deficits that otherwise occurs in MPS I patients. Positive interim data from a Phase I/II trial of RGX-111 were reported in February 2023. RGX-111 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration (FDA). ABOUT REGENXBIO Inc. REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. Since its founding in 2009, REGENXBIO has pioneered the development of AAV Therapeutics, an innovative class of gene therapy medicines. REGENXBIO is advancing a pipeline of AAV Therapeutics for retinal and rare diseases, including ABBV-RGX-314 for the treatment of wet AMD and diabetic retinopathy, being developed in collaboration with AbbVie, RGX-202 for the treatment of Duchenne and RGX-121 for the treatment of MPS II. Thousands of patients have been treated with REGENXBIO's AAV Therapeutic platform, including Novartis' ZOLGENSMA for children with spinal muscular atrophy. Designed to be one-time treatments, AAV Therapeutics have the potential to change the way healthcare is delivered for millions of people. For more information, please visit . ABOUT NIPPON SHINYAKU Based on Nippon Shinyaku's business philosophy, "Helping people lead healthier, happier lives," we aim to be an organization trusted by the community through creating unique medicines that will bring hope to patients and families suffering from illness. Please visit our website () for products or detailed information. FORWARD-LOOKING STATEMENTS This press release includes "forward-looking statements," within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. These statements express a belief, expectation or intention and are generally accompanied by words that convey projected future events or outcomes such as "believe," "may," "will," "estimate," "continue," "anticipate," "assume," "design," "intend," "expect," "could," "plan," "potential," "predict," "seek," "should," "would" or by variations of such words or by similar expressions. The forward-looking statements include statements relating to, among other things, REGENXBIO's future operations, clinical trials, and regulatory plans. REGENXBIO has based these forward-looking statements on its current expectations and assumptions and analyses made by REGENXBIO in light of its experience and its perception of historical trends, current conditions and expected future developments, as well as other factors REGENXBIO believes are appropriate under the circumstances. However, whether actual results and developments will conform with REGENXBIO's expectations and predictions is subject to a number of risks and uncertainties, including the anticipated completion of REGENXBIO's proposed transaction with Nippon Shinyaku, the outcome of REGENXBIO's proposed collaboration with Nippon Shinyaku, whether the milestones contemplated by the proposed transaction will be achieved, the timing of enrollment, commencement and completion and the success of clinical trials conducted by REGENXBIO, its licensees and its partners, the timing of commencement and completion and the success of preclinical studies conducted by REGENXBIO and its development partners, the timely development and launch of new products, the ability to obtain and maintain regulatory approval of product candidates, the ability to obtain and maintain intellectual property protection for product candidates and technology, trends and challenges in the business and markets in which REGENXBIO operates, the size and growth of potential markets for product candidates and the ability to serve those markets, the rate and degree of acceptance of product candidates, and other factors, many of which are beyond the control of REGENXBIO. Refer to the "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of REGENXBIO's Annual Report on Form 10-K for the year ended December 31, 2023, and comparable "risk factors" sections of REGENXBIO's Quarterly Reports on Form 10-Q and other filings, which have been filed with the U.S. Securities and Exchange Commission (SEC) and are available on the SEC's website at . All of the forward-looking statements made in this press release are expressly qualified by the cautionary statements contained or referred to herein. The actual results or developments anticipated may not be realized or, even if substantially realized, they may not have the expected consequences to or effects on REGENXBIO or its businesses or operations. Such statements are not guarantees of future performance and actual results or developments may differ materially from those projected in the forward-looking statements. Readers are cautioned not to rely too heavily on the forward-looking statements contained in this press release. These forward-looking statements speak only as of the date of this press release. Except as required by law, REGENXBIO does not undertake any obligation, and specifically declines any obligation, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. Contacts: Dana Cormack Corporate Communications [email protected] Investors: George E. MacDougall Investor Relations [email protected] SOURCE REGENXBIO Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Orphan DrugFast TrackLicense out/inGene TherapyPriority Review

14 Jan 2025

·www.pharmaceutical-technology.com

JP Morgan 2025: Novartis streamlines its portfolio to drive future growth

Vas Narasimhan, chief executive officer of Novartis AG, speaks during a news conference in Tokyo, Japan. At the JP Morgan Healthcare Conference 2025, Narsimhan talked about how Novartis is optimistic its streamlined portfolio will fuel high growth in the next five years and into the 2030. Photographer: Akio Kon/Bloomberg via Getty Images Positioning itself as a pure-play innovative medicines company, Novartis is bullish about the growth towards 2030 by placing an emphasis on a streamlined portfolio to drive sales, says CEO Vas Narasimhan. Novartis CEO Vas Narasimhan outlined projections for the company’s future at the 2025 JP Morgan Healthcare conference in San Francisco on 14 January. Novartis, he said, expects peak sales of over $3bn for its eight leading branded products which, along with promising growth in China and Japan, which will fuel the company’s revenues throughout the next decade. Chief among these drugs are Cosentyx (secukinumab) and Kisqali (ribociclib succinate), each projected by Novartis to generate peak sales of over $8bn. Alongside these, the company identified four drugs in clinical development, which it expects to drive growth in the 2030s; remibrutinib, OAV101 IT, and ianalumab, all in Phase III trials, and pelacarsen, currently in a Phase II trial (NCT04023552) for aortic valve stenosis. Narasimhan said Novartis expects 15 submission-enabling data readouts in the next two years from their core assets, which will maintain profits in the face of patent expiries for their current blockbuster drugs. Patent expiries of major drugs have been a key issue for big pharma, and is expected to be a major macroeconomic trend affecting the sector this year, as per GlobalData’s State of the BioPharmaceutical Industry 2025 report . GlobalData is the parent company of Pharmaceutical Technology . This in turn has increased the scrutiny on companies’ research and development strategies. Novartis has refocussed its clinical pipeline, moving from 155 active projects in Q3 2021 to 94 in Q3 2024. This, Narasimhan says, has allowed Novartis during this period to invest 49% more full-time equivalent (FTE) per project—the ratio of working hours spent on R&D. The company will continue focusing on four core therapeutic areas-cardiovascular-renal-metabolic, immunology, neuroscience and oncology. The company now expects a compound annual growth rate (CAGR) of over 6% between 2023 and 2028 with roughly similar growth projected for 2029 and beyond. Much of this growth is likely to be found in East Asian markets such as China, where Novartis achieved over 25% growth in sales during 2024, as well as Japan where Narasimhan stated regulatory reforms will aid the company. With an emphasis on artificial intelligence (AI) integration in its R&D and implementation of its fast-to-IND strategy for preclinical assets, Novartis has over 30 potentially high-value new molecular entity (NME) candidates in clinical Phases I-III, which Narasimhan said are set to drive long term revenue growth. The therapeutic areas of focus for the company include immunology, in which Novartis expects over six Phase III and over 10 Phase II readouts in the next five years, and gene therapies, driven by recent acquisitions like Kate Biotherapeutics, which acquired for $1.1bn in November 2024.

AcquisitionPhase 3Phase 2

100 Deals associated with Onasemnogene abeparvovec

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KEGG	Wiki	ATC	Drug Bank
D11559	Onasemnogene abeparvovec	M09AX	DB15528

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
HMN (Hereditary Motor Neuropathy) Proximal Type I	KR	Novartis Gene Therapies, Inc.	28 May 2021
Spinal Muscular Atrophy	US	Novartis Gene Therapies, Inc.	24 May 2019

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Juvenile Spinal Muscular Atrophy	Phase 3	US	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	JP	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	AU	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	BE	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	CA	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	FR	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	IT	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	TW	Novartis Gene Therapies, Inc.	10 Feb 2020
Juvenile Spinal Muscular Atrophy	Phase 3	GB	Novartis Gene Therapies, Inc.	10 Feb 2020
Muscular Atrophy, Spinal, Type II	Phase 3	US	Novartis Gene Therapies, Inc.	10 Feb 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05089656 (STEER, Company_Website) Manual	Phase 3	Muscular Atrophy, Spinal, Type II	-	onasemnogene abeparvovec	hsiwvletwq(rjisabyxnm) = The STEER study met its primary endpoint showing an increase from baseline across the study population in total Hammersmith Functional Motor Scale – Expanded (HFMSE) scores. kpjogmkkut (xzastyhxlj ) Met View more	Positive	30 Dec 2024
NCT05089656 (STEER, Company_Website) Manual	Phase 3	Muscular Atrophy, Spinal, Type II	-	Sham control		Positive	30 Dec 2024
NCT04851873 (SMART, MDA2024) Manual	Phase 3	Spinal Muscular Atrophy	24	onasemnogene abeparvovec	zgiizicxox(kotuqiejey) = gwilketskq evniqcgwtg (slqwnwjywt, 4.0) View more	Positive	04 Mar 2024
NCT05073133 (OFELIA, MDA2024) Manual	Phase 4	Spinal Muscular Atrophy	16	Onasemnogene Abeparvovec	vfdetvldch(kpomdjrrtb) = fyobgpxaxu pywzntdqnu (wypygrlnhc ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec	ewsfhznvly(tssunhsfmq) = Troponin I elevations observed in OA-treated patients in RESTORE appear to be transient and self-limiting and were not associated with clinically concerning cardiac adverse events qcvcznzpmu (eywpklrkzt )	-	03 Mar 2024
MDA2024	Not Applicable	-	-	Onasemnogene abeparvovec	himmmoiqcu(ehkcmjzxsk) = Adverse events were recorded for two patients. None were serious and treatment-related, and event types were consistent with the established safety profile of OA kpwgfwxyep (gekhdbswrc ) View more	-	03 Mar 2024
RESTORE Registry (MDA2024)	Not Applicable	Spinal Muscular Atrophy	31	Onasemnogene abeparvovec	gukrauzibg(tbubrunwtk) = Two deaths were recorded, neither related to treatment afraqybedn (apjdxpyvvi ) View more	Positive	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Onasemnogene abeparvovec (OA)	vcyvcjtnas(hsfyqrvovv) = imypomhbkj fitsqmqjht (clrliehwzy )	-	03 Mar 2024
MDA2024	Not Applicable	Spinal Muscular Atrophy	-	Risdiplam (EVRYSDI)	vcyvcjtnas(hsfyqrvovv) = lnkrqjfnet fitsqmqjht (clrliehwzy )	-	03 Mar 2024
AAN2023	Not Applicable	Spinal Muscular Atrophy SMN2	-	Onasemnogene-abeparvovec	lwrfjfmvrk(bckcgenevm) = hvigplxttw nbrnughegz (faebhbfxmg ) View more	-	25 Apr 2023
NCT04042025 (LT-002, AAN2023)	Phase 3	Spinal Muscular Atrophy	81	Intravenous onasemnogene abeparvovec	mswiqsaill(rcipoevlje) = The most frequently reported TEAEs were gastroenteritis, nasopharyngitis, pneumonia, respiratory distress, and viral infection xkwhduilik (mzuwxfzvyj )	Positive	25 Apr 2023
NCT04042025 (LT-002, AAN2023)	Phase 3	Spinal Muscular Atrophy	81	Intrathecal onasemnogene abeparvovec		Positive	25 Apr 2023
NCT03505099 (SPR1NT, AAN2023)	Phase 3	Spinal Muscular Atrophy SMN2	29	Onasemnogene Abeparvovec	fvusilhino(rquogjvzgo) = buqbzwaxmk sssrqobglh (qixcabvxpl )	-	25 Apr 2023
NCT03505099 (SPR1NT, AAN2023)	Phase 3	Spinal Muscular Atrophy SMN2	29	Onasemnogene Abeparvovec	fvusilhino(rquogjvzgo) = wnmdnbjvzg sssrqobglh (qixcabvxpl )	-	25 Apr 2023

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