Preclinical data generated at Augusta University indicate the drug is well-tolerated and biologically active. Researchers observed that the compound successfully increased fetal hemoglobin levels while simultaneously reducing oxidative stress and the characteristic sickling of red blood cells. These results provide the foundational support for the company to advance the treatment into further clinical development phases.
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vTv Therapeutics Secures FDA Orphan Status for Sickle Cell Candidate
The Food and Drug Administration has granted orphan drug designation to HPPD, an experimental oral treatment for sickle cell disease. This regulatory status provides vTv Therapeutics with significant development incentives, including tax credits and potential market exclusivity for up to seven years for the small molecule compound.
The North Carolina-based biopharmaceutical firm focuses on small molecule candidates targeting chronic conditions, including diabetes. By securing this designation, vTv Therapeutics gains relief from specific regulatory user fees, a move intended to accelerate the path for therapies addressing rare conditions with limited existing treatment options.
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