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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.

Clinical Trials Now
 

Drug Development Updates:

2018

6th December: AVXS-101 to receive expedited regulatory review in the US and Europe

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 

19th October: AveXis Community Update on Submitting their Application for AVXS-101

17th October: SMA UK's submission to NICE's scoping consultation on AVXS-101 treatment for infants with SMA Type 1

27th September: AVEXIS CLINICAL TRIAL FOR INFANTS AGED LESS THAN SIX MONTHS OLD WITH SMA TYPE 1 IN THE UK

19th September: NICE Opens Draft Scoping Consultation for AveXis Gene Therapy for SMA Type 1

3rd May: Novartis Agrees $8.7 Billion Deal to Acquire AveXis

25th January: AveXis Announce Additional Trials of AVXS-101 for SMA
 

2017

15th December: AveXis Announce Phase 1 Trial of AVXS-101 for SMA Type 2

15th December: Summary and comparison of results to date for nusinersen and gene therapy (Avexis-101)

12th October: AveXis to Imminently Initiate AVXS-101 Pivotal Trial in the US

22nd June: AVXS-101 gene therapy manufacturing process streamlined

5th April: AveXis announce results from AVXS-101 Phase I trial

9th February: NEW EUROPEAN PIVOTAL STUDY ANNOUNCED BY AVEXIS
 

2016

7th November: NEW PIVOTAL STUDY ANNOUNCED BY AVEXIS

18th August: AVEXIS PROVIDES UPDATE ON ONGOING AVXS-101 TRIAL

28th July: AVXS-101 RECEIVES “BREAKTHROUGH THERAPY DESIGNATION”

23rd May: AVEXIS PROVIDES UPDATE ON VIRAL GENE THERAPY TRIAL

8th January: AVEXIS COMPLETES PHASE I TRIAL PATIENT ENROLMENT

 

2014

8th October: AVEXIS GENE THERAPY GAINS ORPHAN DRUG STATUS   

30th April: SMN1 GENE THERAPY TRIAL INITIATED IN THE US

15th April: REGENX REACH DEAL WITH AVEXIS TO ADVANCE POTENTIAL SMA GENE THERAPIES

 

2013

23rd October: SMN1 GENE THERAPY KNOWN AS SCAAV9.CB.SMN RECEIVES FAST TRACK STATUS IN THE US