Drugs / Onasemnogene Abeparvovec-xioi
Regulatory milestones approvals, filings & regulatory actions · 4 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Filed spinal muscular atrophy, type 1 3 milestones | |||||
| Filed | EU (EMA) | — | 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 for type 1 SMA” novartis.com ↗ |
| Filed | Japan (PMDA) | — | 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 for type 1 SMA” novartis.com ↗ |
| Filed | US (FDA) | — | 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 for type 1 SMA” novartis.com ↗ |
| Clinical hold arthrogryposis spinal muscular atrophy 1 milestone | |||||
| Clinical hold | US (FDA) | — | arthrogryposis spinal muscular atrophy | 2019-10-30 | “FDA placed a partial hold on AVXS-101 intrathecal clinical trials for SMA patients based on findings in a small pre-clinical animal study” novartis.com ↗ |
Trials 6
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03381729 | Dec 2017 → Nov 2021 | spinal muscular atrophy | Novartis Gene Therapies | Terminated | Mixed Stop: Business |
| Phase 35 trials · 3 met primary | ||||||
| Phase 3 | NCT04042025 | Feb 2020 → Dec 2035 expected | spinal muscular atrophy, type 1, spinal muscular atrophy, type II, spinal muscular atrophy, type III | Novartis Gene Therapies | Active not recruiting | No outcome recorded |
| Phase 3 | NCT03837184 | May 2019 → Jun 2021 | spinal muscular atrophy, type 1 | Novartis Gene Therapies | Completed | No outcome recorded |
| Phase 3 | NCT03461289 | Aug 2018 → Sep 2020 | spinal muscular atrophy | Novartis Gene Therapies | Completed | Met primary |
| Phase 3 | NCT03505099 | Apr 2018 → Jun 2021 | spinal muscular atrophy | Novartis Gene Therapies | Completed | Met primary |
| Phase 3 | NCT03306277 | Oct 2017 → Nov 2019 | spinal muscular atrophy | Novartis Gene Therapies | Completed | Met primary |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 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). |
| 2020-03-24 | 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 |
| 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. |
| 2019-09-19 | 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 24 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 7 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Onasemnogene Abeparvovec-xioi | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03306277 ↗ |
| Known as | AVXS-101 | “We aimed to evaluate the safety and efficacy of onasemnogene abeparvovec (previously known as AVXS-101), a gene therapy delivering the survival motor neuron gene (SMN)” PMID 33743238 ↗ Mar 2021 |
| Action | Restore | “Onasemnogene abeparvovec (previously known as AVXS-101), a gene therapy delivering the survival motor neuron gene (SMN)” PMID 33743238 ↗ Mar 2021 |
| Modality | Gene therapy (AAV / viral vector) | “Self-complementary AAV9 carrying the SMN gene under the control of a hybrid CMV enhancer/chicken-β-actin promoter” NCT03381729 ↗ |
| Route | Intravenous | “Patients received a one-time intravenous infusion of onasemnogene abeparvovec” PMID 33743238 ↗ Mar 2021 |