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Rocket Pharmaceuticals Withdraws FDA Application for Fanconi Anemia Treatment
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Rocket Pharmaceuticals Withdraws FDA Application for Fanconi Anemia Treatment
Oct 3, 2025 5:36 AM

07:59 AM EDT, 10/03/2025 (MT Newswires) -- Rocket Pharmaceuticals ( RCKT ) said Friday it voluntarily withdrew its biologics license application to the US Food and Drug Administration for its investigational mozafancogene autotemcel treatment of Fanconi anemia, a rare inherited disorder.

The withdrawal is mainly due to business reasons and not because of any safety or efficacy concerns, with data to date showing the therapy was "generally well tolerated," the company said in a regulatory filing.

Rocket said it may seek external partners to potentially continue development of the treatment in the future.

The company's shares were down 1.6% in recent premarket activity.

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