AVXS-401, by Novartis Gene Therapies

25/01/2021. AVXS-401 is a gene therapy developed by Novartis Gene Therapies (AveXis, now is part of Novartis). It uses the AAV9 adeno-associated virus (AAV) vector to deliver a functional copy of the FXN gene to restore the production of frataxin in tissues that are severely affected by FA. Early toxicity studies conducted in healthy mice showed that AVXS-401 has good safety and tolerability. Efficacy studies have shown that in a FA mouse model lacking frataxin in the central nervous system, a single intracerebroventricular (ICV) injection of low-dose AVXS-401 can improve behavior and promote glial cell proliferation. In a mouse model lacking frataxin in the heart, AVXS-401 can restore heart function, prolong the median survival by more than 3 times, and prevent the onset of cardiovascular disease. When the therapeutic dose is increased proportionally and administered in non-human primates, AVXS-401 can achieve sustained expression of frataxin in the central nervous system and heart, and the effect is still obvious after 6 months of treatment.

Novartis Gene Therapies gets FDA Orphan Drug Designation in Friedreich's Ataxia

01/21/2021. Novartis Gene Therapies gets FDA Orphan Drug Designation in Friedreich's Ataxia, Novartis Gene Therapies announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation for its "Adeno-associated virus isoform 9 gene vector construct expressing the human Frataxin gene" for the treatment of Friedreich's Ataxia.

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Generic Name:	Adeno-associated virus isoform 9 gene vector construct expressing the human Frataxin gene
Date Designated:	01/21/2021
Orphan Designation:	Treatment of Friedreich`s Ataxia.
Orphan Designation Status:	Designated
FDA Orphan Approval Status:	Not FDA Approved for Orphan Indication
Sponsor:	Novartis Gene Therapies 2275 Half Day Road, Suite 300 Bannockburn, Illinois 60015 United States The sponsor address listed is the last reported by the sponsor to OOPD.

*Exclusivity Protected Indications are shown for approvals from Jan. 1, 2013, to the present.