July 17, 2024

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FDA delays 3-month update on Roktavian gene therapy for hemophilia A



 

FDA delays 3-month update on Roktavian gene therapy for hemophilia A.


Just when BioMarin considered its hemophilia A gene therapy Roktavian ( Valoctogenee roxaparvovec) is approaching the FDA endpoint, the agency has pushed back its approval decision date by three months.

 

On March 6 , BioMarin announced that the submission of the three-year data analysis of its Phase 3 GENEr8-1 study of Roktavian , a gene therapy for hemophilia A, constituted a major amendment due to the substantial amount of additional research data provided by the company, The FDA has decided that it needs more time to review applications and has set a new PDUFA target action date of June 30 , 2023 , three months later than the original date of March 31 .

 

FDA delays 3-month update on Roktavian gene therapy for hemophilia A

 

Roktavian ‘s Phase 3 study included 134 participants and is the longest and largest study of a gene therapy for hemophilia to date.

 

Hank Fuchs , M.D. , President, Global Research and Development, BioMarin said they continued to work closely with the FDA and are grateful for the FDA ‘s active engagement as we seek to bring this important therapy to patients with severe hemophilia A.

In the company’s recent submission on this Three years after the new data on the therapy, the three years of data strengthen the promise of our product and further strengthen our belief that Roctavian has the potential to fundamentally change the standard of care for people with hemophilia A. 

 

Roktavian has had a bumpy ride before, with the gene therapy receiving breakthrough therapy designation back in 2017 .

But in 2020 , BioMarin received a complete response letter from the FDA , which expressed concerns about the drug’s expiration date. In September 2022 , BioMarin resubmitted the latest application.

The FDA completed its pre-license inspection of the manufacturing facility in early December 2022 .

 

Valoctogenee roxaparvovec has been approved by the European Union in August 2022 (trade name Roctavian ), and is the world ‘s first approved gene therapy for hemophilia A.

 

BioMarin previously forecast that Roktavian would generate between $ 100 million and $ 200 million in revenue in 2023 .

 

 

 

 

 

 

FDA delays 3-month update on Roktavian gene therapy for hemophilia A

(source:internet, reference only)


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