Update from Novartis Gene Therapies on treatment for FA  – Ataxia

Update from Novartis Gene Therapies on treatment for FA 

Update from Novartis Gene Therapies on treatment for FA 

Novartis Gene Therapies have announced an update on a gene therapy that they have been developing for Friedreich’s Ataxia (FA) called OAV401.  

Novartis Gene Therapies develops and commercialises gene therapies for rare genetic conditions, such as FA. They have announced that the US Food and Drug Administration (FDA), which is the drug regulatory body in the US, has granted Orphan Drug Designation for OAV401Orphan Drug Designation is a status given to medicines for rare conditions. 

Novartis Gene Therapies are also preparing to submit an Investigational New Drug Application for OAV401 to the FDA by the end of 2021. Approval of this application will allow the start of clinical investigation of OAV401. 


 Please see below the community statement from Novartis Gene Therapies: 

“Dear Friedreich’s Ataxia Community, 

We at Novartis Gene Therapies hope that you and your families have had a good start to 2021, and we would like to begin the year together by expressing that we deeply appreciate your collaboration and partnership and providing you with an update on our OAV401 program for Friedreich’s ataxia (FA). 

In recent months, we have been working closely with the FDA to progress OAV401 toward an Investigational New Drug (IND) application. Today, we are pleased to let you know that we expect to submit an IND by the end of 2021. An active IND will allow us to initiate the clinical investigation of OAV401 for the treatment of FA. 

In other breaking news, our application for Orphan Drug Designation for OAV401 was granted by the FDA. Orphan Drug Designation is intended to facilitate and expedite drug development for rare diseases or conditions for which there are no current treatments available. We are pleased by this recognition from the FDA that we have provided sufficient scientific justification to support the clinical investigation of OAV401 for the treatment of FA. 

Our FA advocacy partners are strong allies who have deepened our understanding of FA and the FA community as we advance our work on promising science with transformative potential for FA. We look forward to continuing to collaborate closely with them and the FA community broadly and commit to providing you with updates on a regular basis as we have more information available to share. 


The Novartis Gene Therapies OAV401 Development Team” 



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