drugset
Drugs / AVXS-101
last change Mar 2025 re-read 3 minutes ago

AVXS-101

also known as onasemnogene abeparvovec

Gene therapy (AAV / viral vector) targets SMN1 via restoration

Developed for
spinal muscular atrophy · spinal muscular atrophy, type 1
Investigated by
AveXis, Inc. · Novartis Gene Therapies · Novartis Pharmaceuticals · United BioSource, LLC

Regulatory milestones approvals, filings & regulatory actions · 1 recorded

MilestoneJurisdictionBrandIndicationDateSentence it was read from
Approved · conditional EU (EMA) Zolgensma — 2020-05-18 europa.eu ↗

Trials 3

today
EU approved 2020
NCT05335876 · Ph 3 NCT02122952 · Ph 1
201420152016201720182019202020212022202320242025202620272028202920302031
PhaseRegistry idDatesIndicationSponsorStatusOutcome
Phase 11 trial · 1 met primary
Phase 1 NCT02122952 May 2014 → Dec 2017 spinal muscular atrophy, type 1 Novartis Gene Therapies Completed Met primary
Phase 31 trial
Phase 3 NCT05335876 Dec 2022 → Apr 2031 expected spinal muscular atrophy Novartis Pharmaceuticals Active not recruiting No outcome recorded
Phase not stated1 trial
— NCT03955679 — spinal muscular atrophy United BioSource, LLC Approved for marketing No outcome recorded

Press releases naming this drug 16 releases

DateIssuerRelease
2025-03-19 Novartis Pharmaceuticals New Novartis Phase III data demonstrate meaningful efficacy and safety results of intrathecal onasemnogene abeparvovec in broad patient population with SMA novartis.com ↗
2024-12-30 Novartis Pharmaceuticals Novartis intrathecal onasemnogene abeparvovec Phase III study meets primary endpoint in children and young adults with SMA novartis.com ↗
2020-09-23 Novartis Pharmaceuticals Novartis Provides Update on AVXS-101 Intrathecal Clinical Development Program novartis.com ↗
2020-06-01 Novartis Pharmaceuticals AveXis Community Statement on the Global Managed Access Program for AVXS-101 novartis.com ↗
2020-03-24 Novartis Pharmaceuticals 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 ↗
2019-12-19 Novartis Pharmaceuticals AveXis Community Statement on Global Managed Access Program for AVXS-101 novartis.com ↗
2019-10-30 Novartis Pharmaceuticals Novartis announces AVXS-101 intrathecal study update novartis.com ↗
2019-08-07 Novartis Pharmaceuticals Novartis stands behind Zolgensma® (onasemnogene abeparvovec-xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy novartis.com ↗
2019-07-25 Novartis Pharmaceuticals AveXis Statement on Access to Zolgensma® (onasemnogene abeparvovec-xioi) novartis.com ↗
2018-12-03 Novartis Pharmaceuticals 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 ↗
2018-04-25 Novartis Pharmaceuticals AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3 novartis.com ↗
2018-04-24 Novartis Pharmaceuticals 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 ↗

Evidence & citations 6 cited values

Every value below carries the sentence it was read from. 5 sources stand behind the page.

FieldValueCited text
Known as AVXS-101 ClinicalTrials.gov intervention name — accepted as the source's own label NCT03955679 ↗
1

NCT02122952 ↗
Known as onasemnogene abeparvovec “This study characterizes motor function responses after early dosing of AVXS-101 (onasemnogene abeparvovec) in gene replacement therapy in infants with severe spinal muscular...” PMID 31277975 ↗ May 2019
1

NCT05335876 ↗
Action Restore “We studied functional replacement of the mutated gene encoding survival motor neuron 1 (SMN1) in this disease.” PMID 29091557 ↗ Nov 2017
Modality Gene therapy (AAV / viral vector) “Fifteen patients with SMA1 received a single dose of intravenous adeno-associated virus serotype 9 carrying SMN complementary DNA encoding the missing SMN protein.” PMID 29091557 ↗ Nov 2017
Route Intravenous “AVXS-101 injected intravenously through a peripheral limb vein.” NCT02122952 ↗
Target SMN1 “We studied functional replacement of the mutated gene encoding survival motor neuron 1 (SMN1) in this disease.” PMID 29091557 ↗ Nov 2017