Drugs / OAV101
OAV101
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed arthrogryposis spinal muscular atrophy 2 milestones | |||||
| Filed | EU (EMA) | — | arthrogryposis spinal muscular atrophy | 2025-07-17 | “Abschluss der Zulassungsanträge für OAV101 IT zur Behandlung von Patienten mit spinaler Muskelatrophie (SMA) in den USA und der EU” novartis.com ↗ “Regulatory submissions for OAV101 IT in patients with spinal muscular atrophy (SMA) were completed in the US and EU” novartis.com ↗ |
| Filed | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2025-07-17 | “Abschluss der Zulassungsanträge für OAV101 IT zur Behandlung von Patienten mit spinaler Muskelatrophie (SMA) in den USA und der EU” novartis.com ↗ “Regulatory submissions for OAV101 IT in patients with spinal muscular atrophy (SMA) were completed in the US and EU” novartis.com ↗ |
| Filed spinal muscular atrophy, type 1 3 milestones | |||||
| Filed | EU (EMA) | Zolgensma | spinal muscular atrophy, type 1 | 2018-10-18 | “Zolgensma[1] (AVXS-101) filed in the US with priority review, in the EU under accelerated assessment, and in Japan with Sakigake designation.” novartis.com ↗ “AVXS-101 simultaneous global submissions in US, EU and Japan[2] for type 1 SMA” novartis.com ↗ |
| Filed | Japan (PMDA) | Zolgensma | spinal muscular atrophy, type 1 | 2018-10-18 | “Zolgensma[1] (AVXS-101) filed in the US with priority review, in the EU under accelerated assessment, and in Japan with Sakigake designation.” novartis.com ↗ “AVXS-101 simultaneous global submissions in US, EU and Japan[2] for type 1 SMA” novartis.com ↗ |
| Filed | US (FDA) | Zolgensma | spinal muscular atrophy, type 1 | 2018-10-18 | “Zolgensma[1] (AVXS-101) filed in the US with priority review, in the EU under accelerated assessment, and in Japan with Sakigake designation.” novartis.com ↗ “AVXS-101 simultaneous global submissions in US, EU and Japan[2] for type 1 SMA” novartis.com ↗ |
Trials 4
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 33 trials · 1 met primary | ||||||
| Phase 3 | NCT05386680 | Jan 2023 → Nov 2024 | spinal muscular atrophy | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 3 | NCT05089656 | Feb 2022 → Nov 2024 | spinal muscular atrophy, type II | Novartis Pharmaceuticals | Completed | Met primary |
| Phase 3 | NCT04851873 | Sep 2021 → Jun 2023 | spinal muscular atrophy | Novartis Pharmaceuticals | Completed | No outcome recorded |
| Phase 41 trial | ||||||
| Phase 4 | NCT05073133 | Nov 2021 → Aug 2023 | spinal muscular atrophy | Novartis Pharmaceuticals | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 31
| Date | Issuer | Release |
|---|---|---|
| 2026-07-02 | Novartis Pharmaceuticals | Regulatory Novartis receives European Commission approval for Itvisma® for spinal muscular atrophy (SMA) novartis.com ↗
With this approval, Itvisma becomes the first and only gene replacement therapy currently approved for this broad SMA population in the European Union. |
| 2026-04-24 | Novartis Pharmaceuticals | Regulatory Novartis receives positive CHMP opinion for Itvisma® for spinal muscular atrophy (SMA) novartis.com ↗ |
| 2025-11-24 | Novartis Pharmaceuticals | Regulatory Novartis receives FDA approval for Itvisma®, the only gene replacement therapy for children two years and older, teens, and adults with spinal muscular atrophy (SMA) novartis.com ↗
Novartis today announced that the US Food and Drug Administration (FDA) has approved Itvisma ® (onasemnogene abeparvovec-brve) for the treatment of children two years and older, teens and adults living with spinal muscular atrophy (SMA) with a confirmed mutation in the survival motor neuron 1 ( SMN1 ) gene, making it the first and only gene replacement therapy available for this broad population. |
| 2025-03-19 | Novartis Pharmaceuticals | Results New Novartis Phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA novartis.com ↗
Novartis announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in a broad population of patients aged two to <18 years with spinal muscular atrophy (SMA). |
| 2024-12-30 | Novartis Pharmaceuticals | Results Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA novartis.com ↗
Novartis today announced positive topline results from the Phase III STEER study. |
| 2024-03-04 | Novartis Pharmaceuticals | Results Novartis presents new data on safety and efficacy of Zolgensma, including maintained and improved motor milestones in older and heavier children with SMA novartis.com ↗
Final data from the SMART study highlight the safety and efficacy profile of Zolgensma in children with SMA weighing ≥ 8.5 kg to ≤ 21 kg, with a mean age of 4.69 years, most of whom (21/24, 87.5%) had discontinued use of another disease modifying therapy at the time of treatment. |
| 2023-03-20 | Novartis Pharmaceuticals | Results Novartis shares Zolgensma long-term data demonstrating sustained durability up to 7.5 years post-dosing; 100% achievement of all assessed milestones in children treated prior to SMA symptom onset novartis.com ↗
Additionally, children with SMA Type 2 treated with investigational intrathecal OAV101 maintained or achieved new development gains |
| 2022-06-17 | Novartis Pharmaceuticals | Results Novartis announces Nature Medicine publication of Zolgensma data demonstrating age-appropriate milestones when treating children with SMA presymptomatically novartis.com ↗
These data demonstrate that, whether they have two or three copies of the SMN2 gene, nearly all children with spinal muscular atrophy (SMA) treated presymptomatically with Zolgensma were able to achieve age-appropriate motor milestones, including sitting independently, standing and walking. |
| 2022-03-14 | Novartis Pharmaceuticals | Results Novartis data again demonstrate age-appropriate development when Zolgensma is used presymptomatically, and post-hoc data reveal SMA Type 1 patients could speak, swallow and maintain airway protection novartis.com ↗
The completed Phase 3 SPR1NT study demonstrated that children with three copies of the SMN2 back-up gene who were treated presymptomatically achieved age-appropriate motor milestones, including standing and walking. |
| 2021-08-03 | Novartis Pharmaceuticals | Regulatory Novartis announces lift of partial clinical trial hold and plans to initiate a new, pivotal Phase 3 study of intrathecal OAV-101 in older patients with SMA novartis.com ↗
Novartis today announced that the U.S. Food and Drug Administration (FDA) has determined that OAV-101 intrathecal (IT) clinical trials for spinal muscular atrophy (SMA) patients may proceed, thereby lifting the partial clinical trial hold initiated in October 2019. |
| 2021-06-18 | Novartis Pharmaceuticals | Results New Zolgensma data demonstrate age-appropriate development when used presymptomatically and rapid, clinically meaningful efficacy in symptomatic children, even those with severe SMA at baseline novartis.com ↗
Novartis today announced data that reinforce the transformational benefit of Zolgensma ® (onasemnogene abeparvovec), an essential, one-time treatment and the only gene therapy for spinal muscular atrophy (SMA). |
| 2021-03-15 | Novartis Pharmaceuticals | Results New Zolgensma data demonstrate age-appropriate development when used early, real-world benefit in older children and durability 5+ years post-treatment novartis.com ↗
Novartis today announced new data that reinforce the transformational benefit of Zolgensma ® (onasemnogene abeparvovec), an essential one-time treatment for spinal muscular atrophy (SMA). |
| 2020-10-01 | Novartis Pharmaceuticals | Results Zolgensma® data including patients with more severe SMA at baseline further demonstrate therapeutic benefit, including prolonged event-free survival, increased motor function and milestone achievement novartis.com ↗
Two-thirds of patients (66.7%) were free of feeding support, an important indicator of stabilization/halting of disease progression |
| 2020-09-23 | Novartis Pharmaceuticals | Regulatory Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program novartis.com ↗
The FDA has acknowledged the potential of AVXS-101 IT in this patient population and recommends a pivotal confirmatory study to supplement the existing STRONG data and further support the regulatory submission for AVXS-101 IT. |
| 2020-05-19 | Novartis Pharmaceuticals | Regulatory AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA) novartis.com ↗
Zolgensma ® (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with up to three copies of the SMN2 gene |
| 2020-03-27 | Novartis Pharmaceuticals | Regulatory AveXis receives positive CHMP opinion for Zolgensma®, the only gene therapy for spinal muscular atrophy (SMA) novartis.com ↗ |
| 2020-03-24 | Novartis Pharmaceuticals | Results Zolgensma® data shows rapid, significant, clinically meaningful benefit in SMA including prolonged event-free survival, motor milestone achievement and durability now up to 5 years post-dosing novartis.com ↗
Interim data from the ongoing SPR1NT study continue to show patients achieved age-appropriate motor milestones when treated with Zolgensma presymptomatically. |
| 2020-03-24 | Novartis Pharmaceuticals | Results AveXis presents AVXS-101 IT data demonstrating remarkable increases in HFMSE scores and a consistent clinically meaningful response in older patients with SMA Type 2 novartis.com ↗
Nearly all patients (92%) in this cohort achieved a clinically meaningful ≥3-point increase during the study period, demonstrating a consistent response following gene therapy |
| 2020-03-19 | Novartis Pharmaceuticals | Regulatory Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma® the only gene therapy for patients with spinal muscular atrophy (SMA) novartis.com ↗
Zolgensma ® (onasemnogene abeparvovec), a one-time administered gene therapy, is approved for the treatment of SMA in patients under the age of two, including those who are pre-symptomatic at diagnosis |
| 2019-10-30 | Novartis Pharmaceuticals | Regulatory Novartis announces AVXS-101 intrathecal study update novartis.com ↗
Basel, October 30, 2019 – Novartis today announced the United States Food & Drug Administration (FDA) placed a partial hold on clinical trials for intrathecal administration of AVXS-101. |
| 2019-10-05 | Novartis Pharmaceuticals | Results AveXis presents updated STRONG data at WMS novartis.com ↗
Older patients (≥ 2 years and < 5 years) achieved a mean increase of 5.9 points from baseline in HFMSE scores, nearly double the clinically meaningful threshold (at a mean duration of follow-up time of 9.3 months) |
| 2019-09-19 | Novartis Pharmaceuticals | Results AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1 novartis.com ↗
Updated results from global STR1VE study demonstrate that Zolgensma ® (onasemnogene abeparvovec-xioi) has significant therapeutic benefit in prolonging event-free survival in SMA Type 1 patients versus natural history |
| 2019-07-18 | Novartis Pharmaceuticals | Regulatory Novartis delivers strong sales, double digit core operating income growth and launches Zolgensma and Piqray in second quarter; sales and profit guidance increased novartis.com ↗
Zolgensma (onasemnogene abeparvovec-xioi) was launched in the US following FDA approval. |
| 2019-05-24 | Novartis Pharmaceuticals | Regulatory AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA) novartis.com ↗
Zolgensma is the first and only gene therapy approved by the FDA for the treatment of SMA, including those who are pre-symptomatic at diagnosis. |
| 2019-05-07 | Novartis Pharmaceuticals | Results New AveXis data at AAN showed long-term durability of Zolgensma® in patients with spinal muscular atrophy (SMA) Type 1 novartis.com ↗
Interim long-term follow-up data showed all enrolled Cohort 2 patients maintained motor function and milestones achieved during the Phase 1 START trial |
| 2019-05-05 | Novartis Pharmaceuticals | Results AveXis presented robust data at AAN demonstrating efficacy of Zolgensma® in broad spectrum of spinal muscular atrophy (SMA) patients novartis.com ↗
Interim data reported for the first time from STRONG in SMA Type 2 showed rapid motor function gains and milestone achievements with intrathecal Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) |
| 2019-04-30 | Novartis Pharmaceuticals | Results New data at AAN reinforce Novartis commitment to transforming the lives of people of all ages who live with neurological conditions novartis.com ↗
Interim data from multiple ongoing clinical studies of Zolgensma ®* (onasemnogene abeparvovec-xioi; AVXS-101) show positive results in the treatment of SMA[1]-[12] |
| 2019-04-16 | Novartis Pharmaceuticals | Results AveXis data reinforce effectiveness of Zolgensma® in treating spinal muscular atrophy (SMA) Type 1 novartis.com ↗
AveXis, a Novartis company, today announced that interim data from its Phase 3 STR1VE trial of Zolgensma ® (onasemnogene abeparvovec-xioi; AVXS-101)[1] in spinal muscular atrophy (SMA) Type 1 showed prolonged event-free survival, an early and rapid increase in CHOP-INTEND scores and significant milestone achievement compared to untreated natural history, consistent with data from the pivotal Phase 1 START trial. |
| 2018-12-03 | Novartis Pharmaceuticals | Regulatory Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1 novartis.com ↗
Novartis today announced that the U.S. Food and Drug Administration (FDA) has accepted the company's Biologics License Application (BLA) for AVXS-101, now known as ZOLGENSMA ® (onasemnogene abeparvovec-xxxx)[1], an investigational gene replacement therapy for the treatment of spinal muscular atrophy (SMA) Type 1. |
| 2018-04-24 | Novartis Pharmaceuticals | Results AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology novartis.com ↗ |
| 2018-03-27 | Novartis Pharmaceuticals | Regulatory AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1 novartis.com ↗
AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) awarded the company's initial product candidate, AVXS-101, SAKIGAKE Designation (SAKIGAKE) for the treatment of spinal muscular atrophy (SMA) Type 1. |
All press releases naming this drug 40 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | OAV101 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05073133 ↗ |
| Known as | AVXS-101 | “Gene Replacement Therapy With Intravenous OAV101 (AVXS-101)” NCT04851873 ↗ |
| Known as | onasemnogene abeparvovec | “STEER ( NCT05089656 ) was a 52-week, phase 3, multicenter, randomized, sham-controlled, double-blind trial evaluating intrathecal onasemnogene abeparvovec (OAV101 IT), a...” PMID 41360993 ↗ Dec 20251“Intrathecal onasemnogene abeparvovec for treatment-experienced patients with spinal muscular atrophy: a phase 3b, open-label trial.” PMID 41360995 ↗ Dec 2025 |
| Action | Restore | “Gene Replacement Therapy” NCT04851873 ↗ |
| Route | Intravenous | “one-time IV infusion” NCT04851873 ↗ |