FDA grants orphan drug status to vTv Therapeutics' sickle cell drug

vTv Therapeutics Inc. (Nasdaq: VTVT) announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation to HPPD (HPP8668), the company's investigational oral Nrf2/Bach1 modulator being studied as a treatment for sickle cell disease.

The designation applies to an early-stage compound that vTv says it intends to advance through partnering discussions, while the company continues to focus primary resources on cadisegliatin, its late-stage type 1 diabetes program.

Preclinical studies conducted at Augusta University using the Townes sickle cell disease mouse model showed that HPPD was orally active and well tolerated, increased fetal hemoglobin, and reduced oxidative stress and red blood cell sickling. Safety and efficacy have not been established in humans.

Paul Sekhri, Chairman, President, and CEO of vTv Therapeutics, said the designation "marks an important inflection point for this program and strengthens vTv's ability to advance strategic partnering discussions for HPPD while continuing to develop cadisegliatin in T1D."

Sickle cell disease affects approximately 100,000 people in the United States, more than 90% of whom are non-Hispanic Black or African American. The disease is characterized by chronic hemolytic anemia, recurrent pain crises, and progressive organ damage. Hydroxyurea is the current standard fetal hemoglobin-inducing therapy, though some patients experience inadequate response or tolerability challenges.

The FDA's Orphan Drug Designation program is intended to encourage development of treatments for rare diseases affecting fewer than 200,000 people in the United States. The designation provides certain development incentives, including tax credits and market exclusivity upon approval.

vTv's lead program, cadisegliatin, holds FDA Breakthrough Therapy designation and is under investigation in a Phase 3 study for type 1 diabetes. The information in this article is based on a company press release.

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