ZOLGENSMA / AVXS-101 (AveXis) Updates
AVXS-101 uses harmless, genetically-engineered viruses to increase SMN protein levels.
In July 2016, it was granted ‘breakthrough status’ in the US to help speed up development. On 18th October 2018 AveXis submitted their regulatory applications for the treatment in the U.S., Europe and Japan for use in infants with SMA Type 1. In October 2018 NICE started its scoping work to see whether this treatment is a ‘correct topic’ they should look at.
Drug Development Updates:
6th November: We attended NICE’s scoping meeting for AVXS-101 advocating for an HST route for the appraisal and speaking up for access. Read our submission
25th January: AveXis Announce Additional Trials of AVXS-101 for SMA
15th December: AveXis Announce Phase 1 Trial of AVXS-101 for SMA Type 2
9th February: NEW EUROPEAN PIVOTAL STUDY ANNOUNCED BY AVEXIS
7th November: NEW PIVOTAL STUDY ANNOUNCED BY AVEXIS
18th August: AVEXIS PROVIDES UPDATE ON ONGOING AVXS-101 TRIAL
8th January: AVEXIS COMPLETES PHASE I TRIAL PATIENT ENROLMENT
8th October: AVEXIS GENE THERAPY GAINS ORPHAN DRUG STATUS
30th April: SMN1 GENE THERAPY TRIAL INITIATED IN THE US