Fanconi Anemia Drug: Promising Advancements Fanconi Ane Drug Undergoes Successful Clinical Trials

Pending positive phase 3 outcomes, FT-2102 could potentially become the first FDA-approved treatment specific for FA. Given the rarity and severity of FA, FDA programs like orphan drug designation and priority review may expedite regulatory review. Submission of a New Drug Application could happen as early as 2025. Approval would represent a major advance in managing this debilitating disease and improving quality of life for FA patients worldwide.

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