Pharma Focus Europe

Unravel Biosciences Secures FDA Orphan Drug Designation for Vorinostat (RVL-001) in Rett Syndrome Treatment

Wednesday, May 29, 2024

Unravel Biosciences, Inc. (Unravel) has just received Orphan Drug Designation from the United States Food and Drug Administration (FDA) for vorinostat (RVL-001) as a potential treatment for Rett Syndrome. Using their proprietary BioNAV™ drug discovery platform, Unravel identified RVL-001 as a promising candidate for addressing Rett Syndrome. Over the past 18 months, Unravel has made significant progress in moving RVL-001 towards clinical trials for Rett patients. Last year, the company received positive feedback in a pre-IND meeting with the FDA regarding its proposed proof-of-concept study for Rett syndrome. In March of this year, Unravel began manufacturing clinical trial materials for RVL-001 to be used in upcoming trials in the US and Colombia. Additionally, last month, Unravel announced a collaboration with Vanderbilt University on a multicenter clinical study in the US to evaluate RVL-001 in patients with Rett syndrome.

Unravel, expressed his satisfaction with the Orphan Drug Designation, stating that it underscores the team's commitment to developing new therapeutic options for patients with rare diseases. He emphasized the significance of this achievement and the recent partnership with Vanderbilt, highlighting Unravel's ability to rapidly prototype drugs and assess their efficacy in patients.

Rett syndrome is a rare disorder characterized by neurogenetic abnormalities that manifest in early childhood and result in severe cognitive, motor, and autonomic impairments. With a global incidence of one in 10,000 female births, there is currently only one FDA-approved treatment, leaving a significant unmet need for novel therapies with meaningful efficacy and favorable safety profiles. In addition to its RVL-001 program, Unravel has initiated development efforts for RVL-002, a molecule targeting Rett syndrome.



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